| patient_id,disease_name,disease_group,trial_id,trial_phase,trial_intervention,eligibility_score,eligible_flag,score_age,score_gene,score_acmg,score_functional,score_safety,score_access,score_geography,score_biomarker,primary_endpoint |
| HLT011-000001,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8575,1,1.0,1.0,1.0,0.0,1.0,1.0,0.946,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000002,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4742,0,0.4,0.0,1.0,0.0,1.0,1.0,0.847,1.0,Aortic root z-score change at 36 months |
| HLT011-000003,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7769,1,1.0,0.0,1.0,1.0,1.0,1.0,0.957,1.0,Aortic root z-score change at 36 months |
| HLT011-000004,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7771,1,1.0,1.0,1.0,0.0,1.0,0.7,0.92,1.0,TKV change from baseline at 36 months |
| HLT011-000005,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8705,1,0.4,1.0,1.0,1.0,1.0,1.0,0.987,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000006,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.481,0,1.0,0.0,1.0,0.0,0.0,0.7,0.639,1.0,Aortic root z-score change at 36 months |
| HLT011-000007,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7353,1,1.0,0.0,1.0,1.0,1.0,1.0,0.825,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000008,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8262,1,1.0,1.0,0.3,1.0,1.0,0.2,0.958,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000009,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8115,1,1.0,1.0,1.0,0.0,1.0,1.0,0.571,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000010,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6038,1,1.0,0.0,1.0,0.0,1.0,0.7,0.646,1.0,Aortic root z-score change at 36 months |
| HLT011-000011,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.856,1.0,Aortic root z-score change at 36 months |
| HLT011-000012,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7517,1,1.0,0.0,1.0,1.0,1.0,1.0,0.989,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000013,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.825,1.0,TKV change from baseline at 36 months |
| HLT011-000014,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7624,1,1.0,0.0,1.0,1.0,1.0,1.0,0.945,1.0,MLMT performance score improvement at 1 year |
| HLT011-000015,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7231,1,1.0,0.0,1.0,1.0,1.0,1.0,0.961,1.0,Aortic root z-score change at 36 months |
| HLT011-000016,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8092,1,1.0,1.0,0.0,1.0,1.0,1.0,0.939,1.0,6MWT distance change at 48 weeks |
| HLT011-000017,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8626,1,0.4,1.0,1.0,1.0,1.0,0.7,0.778,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000018,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7421,1,1.0,0.0,1.0,1.0,1.0,1.0,0.785,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000019,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5287,0,1.0,0.0,0.0,1.0,1.0,0.7,0.876,1.0,MLMT performance score improvement at 1 year |
| HLT011-000020,Wiskott-Aldrich Syndrome,Rare immunological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5965,1,1.0,0.0,1.0,1.0,0.0,0.2,0.966,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000021,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6863,1,1.0,0.0,1.0,1.0,0.0,1.0,0.989,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000022,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2727,0,0.0,0.0,0.3,0.0,1.0,1.0,0.183,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000023,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7383,1,1.0,0.0,1.0,1.0,1.0,1.0,0.963,1.0,MLMT performance score improvement at 1 year |
| HLT011-000024,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.581,1,1.0,0.0,1.0,0.0,1.0,1.0,0.0,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000025,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.878,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000026,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6094,1,0.4,1.0,0.0,1.0,0.0,0.7,0.948,1.0,6MWT distance change at 48 weeks |
| HLT011-000027,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8125,1,1.0,0.0,1.0,1.0,1.0,1.0,0.943,1.0,Aortic root z-score change at 36 months |
| HLT011-000028,Pompe Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4812,0,0.4,0.0,1.0,0.0,1.0,0.7,0.846,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000029,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6904,1,1.0,1.0,0.3,0.0,1.0,1.0,0.86,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000030,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6626,1,0.0,1.0,1.0,0.0,1.0,0.7,0.968,1.0,TKV change from baseline at 36 months |
| HLT011-000031,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6479,1,1.0,1.0,0.0,0.0,1.0,0.5,0.98,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000032,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.352,0,0.4,0.0,0.0,1.0,0.0,1.0,0.849,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000033,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5164,0,1.0,0.0,0.0,1.0,1.0,1.0,0.719,1.0,Motor milestone achievement at 14 months |
| HLT011-000034,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5949,1,0.4,1.0,0.3,0.0,1.0,1.0,0.987,1.0,Aortic root z-score change at 36 months |
| HLT011-000035,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7664,1,1.0,0.0,1.0,1.0,1.0,1.0,0.896,1.0,Aortic root z-score change at 36 months |
| HLT011-000036,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9199,1,0.4,1.0,1.0,1.0,1.0,0.7,0.985,1.0,6MWT distance change at 48 weeks |
| HLT011-000037,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8575,1,1.0,1.0,1.0,0.0,1.0,1.0,0.706,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000038,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3447,0,1.0,0.0,0.0,0.0,1.0,0.2,0.889,1.0,Aortic root z-score change at 36 months |
| HLT011-000039,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8432,1,1.0,1.0,1.0,0.0,1.0,1.0,0.541,1.0,6MWT distance change at 48 weeks |
| HLT011-000040,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8271,1,0.0,1.0,1.0,1.0,1.0,0.5,0.786,1.0,TKV change from baseline at 36 months |
| HLT011-000041,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4268,0,1.0,0.0,0.3,0.0,1.0,0.2,0.742,1.0,Aortic root z-score change at 36 months |
| HLT011-000042,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.726,1,1.0,0.0,1.0,1.0,1.0,0.2,0.396,1.0,MLMT performance score improvement at 1 year |
| HLT011-000043,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.952,1.0,TKV change from baseline at 36 months |
| HLT011-000044,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7315,1,1.0,0.0,1.0,1.0,1.0,0.7,0.716,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000045,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7341,1,1.0,0.0,1.0,1.0,1.0,0.7,0.582,1.0,MLMT performance score improvement at 1 year |
| HLT011-000046,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.8842,1,1.0,1.0,1.0,1.0,0.0,0.2,0.91,1.0,MLMT performance score improvement at 1 year |
| HLT011-000047,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6041,1,0.4,0.0,1.0,1.0,1.0,0.7,0.994,1.0,Aortic root z-score change at 36 months |
| HLT011-000048,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9738,1,1.0,1.0,1.0,1.0,1.0,0.7,0.845,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000049,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7996,1,1.0,1.0,1.0,0.0,1.0,0.2,0.856,1.0,TKV change from baseline at 36 months |
| HLT011-000050,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8244,1,1.0,1.0,1.0,0.0,1.0,1.0,0.785,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000051,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7897,1,1.0,1.0,0.0,1.0,1.0,1.0,0.932,1.0,6MWT distance change at 48 weeks |
| HLT011-000052,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8812,1,1.0,1.0,1.0,0.0,1.0,0.7,0.944,1.0,Enzyme activity normalization at 12 months |
| HLT011-000053,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9271,1,1.0,1.0,1.0,1.0,1.0,0.2,0.501,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000054,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8768,1,1.0,1.0,1.0,0.0,1.0,1.0,0.926,1.0,TKV change from baseline at 36 months |
| HLT011-000055,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7668,1,1.0,1.0,1.0,0.0,1.0,0.2,0.975,1.0,TKV change from baseline at 36 months |
| HLT011-000056,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9814,1,1.0,1.0,1.0,1.0,1.0,1.0,0.716,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000057,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5141,0,0.4,0.0,1.0,0.0,1.0,1.0,0.975,1.0,Aortic root z-score change at 36 months |
| HLT011-000058,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7269,1,0.4,1.0,0.0,1.0,1.0,1.0,0.977,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000059,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8876,1,1.0,1.0,1.0,1.0,0.0,0.2,0.942,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000060,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.867,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000061,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8574,1,1.0,1.0,1.0,1.0,0.0,0.2,0.996,1.0,TKV change from baseline at 36 months |
| HLT011-000062,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5942,1,1.0,0.0,0.3,1.0,1.0,1.0,0.937,1.0,Aortic root z-score change at 36 months |
| HLT011-000063,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7804,1,0.4,1.0,0.3,1.0,1.0,0.7,0.89,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000064,Epidermolysis Bullosa,Rare dermatological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7411,1,1.0,0.0,1.0,1.0,1.0,1.0,0.894,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000065,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.2577,0,0.0,0.0,1.0,0.0,0.0,0.2,0.266,1.0,TKV change from baseline at 36 months |
| HLT011-000066,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4258,0,0.0,1.0,0.0,0.0,1.0,0.2,0.725,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000067,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8023,1,0.0,1.0,1.0,1.0,1.0,0.7,0.729,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000068,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.977,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000069,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8214,1,0.0,1.0,1.0,1.0,1.0,0.2,0.972,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000070,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5981,1,1.0,0.0,0.3,1.0,1.0,0.2,0.98,1.0,Aortic root z-score change at 36 months |
| HLT011-000071,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9827,1,1.0,1.0,1.0,1.0,1.0,1.0,0.85,1.0,6MWT distance change at 48 weeks |
| HLT011-000072,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.648,1,0.4,0.0,1.0,1.0,1.0,0.7,0.843,1.0,Aortic root z-score change at 36 months |
| HLT011-000073,Li-Fraumeni Syndrome,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7361,1,1.0,0.0,1.0,1.0,1.0,1.0,0.845,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000074,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.992,1.0,Motor milestone achievement at 14 months |
| HLT011-000075,Niemann-Pick Type C,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7986,1,1.0,1.0,0.3,1.0,1.0,0.2,0.681,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000076,Friedreich Ataxia,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4283,0,0.0,0.0,0.3,1.0,1.0,0.5,0.971,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000077,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9868,1,1.0,1.0,1.0,1.0,1.0,1.0,0.942,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000078,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9479,1,1.0,1.0,1.0,1.0,1.0,0.7,0.861,1.0,TKV change from baseline at 36 months |
| HLT011-000079,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6747,1,0.0,1.0,0.3,1.0,1.0,0.2,0.403,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000080,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9518,1,1.0,1.0,1.0,1.0,1.0,0.2,0.931,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000081,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9664,1,1.0,1.0,1.0,1.0,1.0,0.2,0.934,1.0,TKV change from baseline at 36 months |
| HLT011-000082,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7626,1,1.0,0.0,1.0,1.0,1.0,1.0,0.938,1.0,Aortic root z-score change at 36 months |
| HLT011-000083,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6055,1,1.0,0.0,1.0,0.0,1.0,1.0,0.735,1.0,MLMT performance score improvement at 1 year |
| HLT011-000084,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6398,1,0.4,1.0,0.3,0.0,1.0,1.0,0.995,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000085,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9614,1,1.0,1.0,1.0,1.0,1.0,0.2,0.88,1.0,Enzyme activity normalization at 12 months |
| HLT011-000086,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8449,1,1.0,1.0,0.3,1.0,1.0,1.0,0.922,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000087,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9755,1,1.0,1.0,1.0,1.0,1.0,0.5,0.929,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000088,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6831,1,1.0,0.0,1.0,1.0,1.0,0.7,0.859,1.0,Aortic root z-score change at 36 months |
| HLT011-000089,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7449,1,0.4,1.0,1.0,1.0,0.0,0.5,0.942,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000090,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5776,0,0.0,1.0,0.0,1.0,1.0,0.2,0.975,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000091,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7834,1,1.0,1.0,1.0,0.0,1.0,0.2,0.843,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000092,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8265,1,1.0,1.0,1.0,0.0,1.0,0.2,0.883,1.0,TKV change from baseline at 36 months |
| HLT011-000093,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.712,1,1.0,0.0,1.0,1.0,1.0,1.0,0.64,1.0,Aortic root z-score change at 36 months |
| HLT011-000094,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.497,0,0.4,0.0,1.0,0.0,1.0,1.0,0.944,1.0,Aortic root z-score change at 36 months |
| HLT011-000095,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9124,1,1.0,1.0,1.0,1.0,0.0,1.0,0.113,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000096,Wiskott-Aldrich Syndrome,Rare immunological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.3586,0,0.4,0.0,0.3,0.0,1.0,0.2,0.949,1.0,6MWT distance change at 48 weeks |
| HLT011-000097,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.3432,0,1.0,0.0,0.0,0.0,1.0,0.5,0.707,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000098,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.4278,0,1.0,0.0,0.3,0.0,1.0,1.0,0.877,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000099,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7221,1,0.4,1.0,1.0,0.0,1.0,0.7,0.476,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000100,Prader-Willi Syndrome,Chromosomal,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3795,0,0.0,0.0,0.3,1.0,0.0,1.0,0.81,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000101,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5481,0,1.0,0.0,0.0,1.0,1.0,0.7,0.903,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000102,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8162,1,0.4,1.0,1.0,1.0,0.0,1.0,0.969,1.0,TKV change from baseline at 36 months |
| HLT011-000103,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5388,0,0.0,1.0,0.3,0.0,1.0,1.0,0.886,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000104,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6938,1,1.0,0.0,1.0,1.0,1.0,0.7,0.971,1.0,6MWT distance change at 48 weeks |
| HLT011-000105,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.943,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000106,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6689,1,0.0,1.0,1.0,0.0,1.0,0.7,0.893,1.0,TKV change from baseline at 36 months |
| HLT011-000107,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5907,1,1.0,0.0,1.0,0.0,1.0,0.7,0.858,1.0,Aortic root z-score change at 36 months |
| HLT011-000108,Friedreich Ataxia,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6739,1,1.0,0.0,1.0,1.0,0.0,0.7,0.595,1.0,Motor milestone achievement at 14 months |
| HLT011-000109,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6008,1,1.0,1.0,0.0,0.0,1.0,0.7,0.0,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000110,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7181,1,1.0,0.0,1.0,1.0,1.0,1.0,0.961,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000111,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7529,1,1.0,0.0,1.0,1.0,1.0,1.0,0.918,1.0,Aortic root z-score change at 36 months |
| HLT011-000112,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.866,1.0,6MWT distance change at 48 weeks |
| HLT011-000113,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5739,0,1.0,0.0,0.0,1.0,1.0,1.0,0.963,1.0,Motor milestone achievement at 14 months |
| HLT011-000114,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5956,0,0.0,0.0,1.0,1.0,1.0,0.7,0.898,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000115,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.99,1.0,6MWT distance change at 48 weeks |
| HLT011-000116,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6178,1,1.0,0.0,1.0,1.0,0.0,0.7,0.966,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000117,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7186,1,1.0,0.0,1.0,1.0,1.0,1.0,0.879,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000118,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6869,1,0.0,1.0,1.0,0.0,1.0,1.0,0.952,1.0,TKV change from baseline at 36 months |
| HLT011-000119,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.705,1,1.0,0.0,1.0,1.0,1.0,1.0,0.877,1.0,Aortic root z-score change at 36 months |
| HLT011-000120,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4389,0,0.0,0.0,1.0,0.0,1.0,0.5,0.661,1.0,TKV change from baseline at 36 months |
| HLT011-000121,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.1875,0,0.0,0.0,0.0,0.0,1.0,0.7,0.789,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000122,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7741,1,0.4,1.0,1.0,1.0,0.0,1.0,0.819,1.0,TKV change from baseline at 36 months |
| HLT011-000123,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.657,1,1.0,0.0,1.0,1.0,0.0,1.0,0.921,1.0,Enzyme activity normalization at 12 months |
| HLT011-000124,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6273,1,1.0,0.0,0.3,1.0,1.0,1.0,0.74,1.0,Aortic root z-score change at 36 months |
| HLT011-000125,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7625,1,0.0,1.0,1.0,1.0,0.0,0.2,0.486,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000126,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6321,1,0.0,1.0,0.3,0.0,1.0,0.7,0.89,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000127,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5496,0,0.0,0.0,1.0,1.0,1.0,0.7,0.71,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000128,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8328,1,0.4,1.0,1.0,1.0,1.0,0.7,0.958,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000129,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.898,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000130,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8631,1,0.4,1.0,1.0,1.0,1.0,0.7,0.649,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000131,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9713,1,1.0,1.0,1.0,1.0,1.0,0.7,0.858,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000132,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6426,1,1.0,0.0,1.0,1.0,0.0,1.0,0.893,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000133,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4305,0,0.0,0.0,0.0,1.0,1.0,1.0,0.949,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000134,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6152,1,1.0,0.0,1.0,1.0,0.0,1.0,0.0,1.0,Motor milestone achievement at 14 months |
| HLT011-000135,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5151,0,0.4,1.0,0.0,0.0,1.0,1.0,0.541,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000136,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9375,1,1.0,1.0,1.0,1.0,1.0,1.0,0.59,1.0,Enzyme activity normalization at 12 months |
| HLT011-000137,Turner Syndrome,Chromosomal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7445,1,1.0,0.0,1.0,1.0,1.0,1.0,0.869,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000138,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6485,1,1.0,0.0,1.0,0.0,1.0,1.0,0.849,1.0,Aortic root z-score change at 36 months |
| HLT011-000139,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.898,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000140,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7443,1,1.0,0.0,1.0,1.0,1.0,0.2,0.894,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000141,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6961,1,1.0,0.0,1.0,1.0,1.0,0.2,0.768,1.0,Aortic root z-score change at 36 months |
| HLT011-000142,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8134,1,0.0,1.0,1.0,1.0,1.0,1.0,0.964,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000143,Down Syndrome,Chromosomal,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7105,1,1.0,0.0,1.0,1.0,1.0,0.7,0.81,1.0,6MWT distance change at 48 weeks |
| HLT011-000144,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6931,1,1.0,1.0,0.0,0.0,1.0,0.5,0.973,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000145,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5837,1,1.0,0.0,1.0,1.0,0.0,0.2,0.679,1.0,Aortic root z-score change at 36 months |
| HLT011-000146,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5606,0,0.0,1.0,0.0,1.0,0.0,1.0,0.514,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000147,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5808,0,1.0,0.0,0.3,1.0,1.0,1.0,0.929,1.0,Motor milestone achievement at 14 months |
| HLT011-000148,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7173,1,0.4,1.0,0.0,1.0,1.0,0.5,0.977,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000149,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7602,1,1.0,0.0,1.0,1.0,1.0,1.0,0.956,1.0,Aortic root z-score change at 36 months |
| HLT011-000150,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8109,1,1.0,1.0,1.0,0.0,1.0,0.5,0.52,1.0,Enzyme activity normalization at 12 months |
| HLT011-000151,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6371,1,1.0,0.0,1.0,1.0,0.0,1.0,0.611,1.0,MLMT performance score improvement at 1 year |
| HLT011-000152,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6123,1,1.0,0.0,1.0,0.0,1.0,1.0,0.964,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000153,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5214,0,1.0,1.0,0.0,0.0,0.0,0.7,0.9,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000154,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5217,0,0.0,1.0,0.3,0.0,1.0,1.0,0.95,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000155,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.793,1,1.0,1.0,1.0,0.0,1.0,0.7,0.777,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000156,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8882,1,1.0,1.0,1.0,1.0,0.0,0.7,0.833,1.0,TKV change from baseline at 36 months |
| HLT011-000157,Friedreich Ataxia,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5744,1,0.4,0.0,1.0,1.0,0.0,0.2,0.969,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000158,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5627,1,0.0,1.0,0.3,0.0,1.0,1.0,0.931,1.0,TKV change from baseline at 36 months |
| HLT011-000159,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9611,1,1.0,1.0,1.0,1.0,1.0,0.2,0.888,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000160,Epidermolysis Bullosa,Rare dermatological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7561,1,1.0,0.0,1.0,1.0,1.0,0.7,0.297,1.0,Motor milestone achievement at 14 months |
| HLT011-000161,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.856,1,1.0,1.0,1.0,0.0,1.0,1.0,0.981,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000162,Down Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4198,0,0.0,0.0,1.0,0.0,1.0,0.7,0.804,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000163,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.507,1.0,Motor milestone achievement at 14 months |
| HLT011-000164,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.856,1,0.0,1.0,1.0,1.0,1.0,1.0,0.771,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000165,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6613,1,0.0,1.0,0.0,1.0,1.0,0.2,0.908,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000166,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.926,1.0,Aortic root z-score change at 36 months |
| HLT011-000167,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.572,0,1.0,0.0,0.3,1.0,1.0,1.0,0.956,1.0,MLMT performance score improvement at 1 year |
| HLT011-000168,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7848,1,1.0,1.0,1.0,0.0,1.0,0.7,0.596,1.0,MLMT performance score improvement at 1 year |
| HLT011-000169,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7867,1,1.0,0.0,1.0,1.0,1.0,0.7,0.901,1.0,Motor milestone achievement at 14 months |
| HLT011-000170,Down Syndrome,Chromosomal,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5429,0,1.0,0.0,0.0,1.0,1.0,1.0,0.941,1.0,Motor milestone achievement at 14 months |
| HLT011-000171,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9709,1,1.0,1.0,1.0,1.0,1.0,0.7,0.812,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000172,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5375,0,0.0,1.0,0.0,0.0,1.0,1.0,0.787,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000173,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7438,1,1.0,1.0,0.0,1.0,1.0,0.5,0.9,1.0,Motor milestone achievement at 14 months |
| HLT011-000174,Down Syndrome,Chromosomal,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2129,0,0.0,0.0,0.3,0.0,0.0,1.0,0.454,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000175,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8414,1,1.0,1.0,0.3,1.0,1.0,1.0,0.775,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000176,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.612,1,1.0,0.0,1.0,1.0,0.0,0.2,0.806,1.0,Enzyme activity normalization at 12 months |
| HLT011-000177,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.558,1,1.0,0.0,1.0,0.0,1.0,0.2,0.829,1.0,Aortic root z-score change at 36 months |
| HLT011-000178,Alagille Syndrome,Rare hepatic,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.618,1,1.0,0.0,1.0,0.0,1.0,0.7,0.592,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000179,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5649,1,1.0,0.0,1.0,0.0,1.0,0.2,0.806,1.0,Enzyme activity normalization at 12 months |
| HLT011-000180,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5564,1,1.0,1.0,0.0,0.0,0.0,0.7,0.97,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000181,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7143,1,1.0,0.0,1.0,1.0,1.0,0.2,0.926,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000182,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.2116,0,0.0,0.0,0.0,0.0,1.0,0.7,0.963,1.0,Motor milestone achievement at 14 months |
| HLT011-000183,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6532,1,0.4,1.0,0.0,1.0,1.0,0.5,0.992,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000184,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4255,0,0.0,0.0,1.0,0.0,1.0,0.7,0.919,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000185,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.897,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000186,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6464,1,0.0,1.0,1.0,0.0,1.0,0.7,0.94,1.0,TKV change from baseline at 36 months |
| HLT011-000187,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7447,1,0.4,1.0,1.0,0.0,1.0,1.0,0.461,1.0,Aortic root z-score change at 36 months |
| HLT011-000188,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7803,1,1.0,1.0,1.0,0.0,1.0,0.2,0.229,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000189,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9692,1,1.0,1.0,1.0,1.0,1.0,0.5,0.968,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000190,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6794,1,0.0,1.0,0.0,1.0,1.0,1.0,0.976,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000191,Down Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.4036,0,0.4,0.0,1.0,0.0,0.0,0.5,0.914,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000192,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.914,1.0,Motor milestone achievement at 14 months |
| HLT011-000193,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7901,1,1.0,1.0,0.0,1.0,1.0,1.0,0.825,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000194,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5434,0,1.0,0.0,1.0,0.0,1.0,0.2,0.874,1.0,MLMT performance score improvement at 1 year |
| HLT011-000195,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6086,1,0.4,1.0,0.3,0.0,1.0,1.0,0.842,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000196,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9691,1,1.0,1.0,1.0,1.0,1.0,0.2,0.794,1.0,6MWT distance change at 48 weeks |
| HLT011-000197,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5508,0,0.0,0.0,1.0,1.0,1.0,0.7,0.845,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000198,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5307,1,1.0,0.0,0.0,1.0,1.0,0.7,0.666,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000199,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6933,1,0.4,1.0,0.0,1.0,1.0,0.5,0.883,1.0,6MWT distance change at 48 weeks |
| HLT011-000200,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6054,1,1.0,0.0,0.0,1.0,1.0,1.0,0.969,1.0,Motor milestone achievement at 14 months |
| HLT011-000201,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8611,1,1.0,1.0,1.0,1.0,0.0,0.7,0.958,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000202,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7397,1,1.0,0.0,1.0,1.0,1.0,1.0,0.944,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000203,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4694,0,0.0,1.0,0.0,1.0,0.0,0.2,0.844,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000204,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8541,1,0.4,1.0,1.0,1.0,1.0,0.2,0.795,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000205,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.666,1,0.4,1.0,1.0,0.0,0.0,1.0,0.885,1.0,Enzyme activity normalization at 12 months |
| HLT011-000206,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8119,1,1.0,1.0,0.0,1.0,1.0,0.5,0.858,1.0,Motor milestone achievement at 14 months |
| HLT011-000207,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9368,1,1.0,1.0,1.0,1.0,1.0,0.5,0.288,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000208,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7293,1,1.0,1.0,1.0,0.0,0.0,1.0,0.965,1.0,TKV change from baseline at 36 months |
| HLT011-000209,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7704,1,1.0,1.0,1.0,0.0,1.0,0.7,0.895,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000210,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6917,1,0.0,1.0,1.0,0.0,1.0,1.0,0.886,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000211,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7258,1,0.4,1.0,1.0,0.0,1.0,1.0,0.921,1.0,TKV change from baseline at 36 months |
| HLT011-000212,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7757,1,0.4,1.0,0.0,1.0,1.0,1.0,0.979,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000213,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9697,1,1.0,1.0,1.0,1.0,1.0,0.7,0.919,1.0,6MWT distance change at 48 weeks |
| HLT011-000214,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.969,1,1.0,1.0,1.0,1.0,1.0,1.0,0.844,1.0,MLMT performance score improvement at 1 year |
| HLT011-000215,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5549,1,0.4,1.0,0.0,0.0,1.0,1.0,0.923,1.0,TKV change from baseline at 36 months |
| HLT011-000216,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9306,1,1.0,1.0,1.0,1.0,1.0,0.7,0.843,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000217,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8007,1,1.0,1.0,1.0,0.0,1.0,1.0,0.824,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000218,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8928,1,1.0,1.0,1.0,0.0,1.0,0.7,0.697,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000219,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7107,1,1.0,0.0,1.0,1.0,1.0,1.0,0.977,1.0,MLMT performance score improvement at 1 year |
| HLT011-000220,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5413,0,1.0,0.0,1.0,0.0,1.0,0.7,0.541,1.0,Aortic root z-score change at 36 months |
| HLT011-000221,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9412,1,1.0,1.0,1.0,1.0,0.0,0.7,0.938,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000222,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8194,1,1.0,0.0,1.0,1.0,1.0,0.7,0.807,1.0,Aortic root z-score change at 36 months |
| HLT011-000223,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9575,1,1.0,1.0,1.0,1.0,1.0,1.0,0.754,1.0,TKV change from baseline at 36 months |
| HLT011-000224,Turner Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3532,0,0.0,0.0,0.3,1.0,0.0,0.7,0.833,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000225,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9005,1,1.0,1.0,1.0,0.0,1.0,1.0,0.956,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000226,Down Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4488,0,0.0,0.0,1.0,1.0,0.0,0.5,0.947,1.0,TKV change from baseline at 36 months |
| HLT011-000227,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9594,1,1.0,1.0,1.0,1.0,1.0,0.2,0.96,1.0,Enzyme activity normalization at 12 months |
| HLT011-000228,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8719,1,0.0,1.0,1.0,1.0,1.0,1.0,0.953,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000229,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5873,1,1.0,0.0,0.3,1.0,1.0,0.2,0.876,1.0,TKV change from baseline at 36 months |
| HLT011-000230,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7133,1,0.4,1.0,1.0,0.0,1.0,0.7,0.218,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000231,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6508,1,0.4,1.0,1.0,0.0,0.0,1.0,0.827,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000232,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5562,1,1.0,0.0,1.0,0.0,1.0,1.0,0.915,1.0,Aortic root z-score change at 36 months |
| HLT011-000233,Angelman Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5899,1,0.0,0.0,1.0,1.0,1.0,1.0,0.928,1.0,TKV change from baseline at 36 months |
| HLT011-000234,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5752,0,0.0,0.0,1.0,1.0,1.0,0.5,0.608,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000235,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6118,1,1.0,0.0,1.0,0.0,1.0,1.0,0.923,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000236,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.607,1.0,MLMT performance score improvement at 1 year |
| HLT011-000237,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4969,0,1.0,0.0,1.0,0.0,0.0,1.0,0.74,1.0,Aortic root z-score change at 36 months |
| HLT011-000238,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8383,1,1.0,1.0,0.3,1.0,1.0,0.2,0.991,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000239,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8596,1,1.0,1.0,0.3,1.0,1.0,1.0,0.932,1.0,Enzyme activity normalization at 12 months |
| HLT011-000240,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9773,1,1.0,1.0,1.0,1.0,1.0,0.5,0.909,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000241,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5657,0,1.0,0.0,1.0,0.0,1.0,1.0,0.577,1.0,MLMT performance score improvement at 1 year |
| HLT011-000242,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9944,1,1.0,1.0,1.0,1.0,1.0,0.7,0.897,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000243,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9873,1,1.0,1.0,1.0,1.0,1.0,1.0,0.675,1.0,Motor milestone achievement at 14 months |
| HLT011-000244,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7493,1,0.0,1.0,1.0,0.0,1.0,0.7,0.95,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000245,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4837,0,0.0,0.0,1.0,0.0,1.0,0.7,0.951,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000246,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6183,1,1.0,0.0,1.0,1.0,0.0,1.0,0.705,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000247,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5602,0,0.0,0.0,1.0,1.0,1.0,1.0,0.786,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000248,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6221,1,1.0,0.0,0.3,1.0,1.0,0.5,0.878,1.0,Aortic root z-score change at 36 months |
| HLT011-000249,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7454,1,1.0,1.0,1.0,0.0,0.0,0.7,0.985,1.0,Aortic root z-score change at 36 months |
| HLT011-000250,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6138,1,1.0,0.0,1.0,0.0,1.0,1.0,0.978,1.0,Aortic root z-score change at 36 months |
| HLT011-000251,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7434,1,0.4,1.0,1.0,1.0,0.0,0.7,0.926,1.0,TKV change from baseline at 36 months |
| HLT011-000252,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.2757,0,0.4,0.0,0.3,0.0,0.0,1.0,0.735,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000253,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6747,1,1.0,0.0,1.0,1.0,1.0,0.7,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-000254,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7737,1,1.0,1.0,0.0,1.0,1.0,1.0,0.596,1.0,Motor milestone achievement at 14 months |
| HLT011-000255,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8753,1,1.0,1.0,1.0,1.0,0.0,0.2,0.981,1.0,Motor milestone achievement at 14 months |
| HLT011-000256,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7669,1,0.4,1.0,0.3,1.0,1.0,1.0,0.844,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000257,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5756,0,1.0,0.0,1.0,0.0,1.0,0.7,0.88,1.0,MLMT performance score improvement at 1 year |
| HLT011-000258,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7432,1,0.4,1.0,1.0,0.0,1.0,0.7,0.943,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000259,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.55,1,0.4,0.0,1.0,1.0,0.0,1.0,0.918,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000260,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4005,0,0.0,0.0,0.0,1.0,1.0,0.2,0.612,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000261,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9192,1,1.0,1.0,1.0,1.0,1.0,1.0,0.875,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000262,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7421,1,1.0,0.0,1.0,1.0,1.0,1.0,0.974,1.0,MLMT performance score improvement at 1 year |
| HLT011-000263,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4017,0,0.0,0.0,0.0,1.0,1.0,1.0,0.881,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000264,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8441,1,1.0,1.0,1.0,0.0,1.0,0.7,0.908,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000265,Down Syndrome,Chromosomal,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5539,1,1.0,0.0,0.3,1.0,1.0,1.0,0.888,1.0,Enzyme activity normalization at 12 months |
| HLT011-000266,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9965,1,1.0,1.0,1.0,1.0,1.0,0.7,0.955,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000267,Rett Syndrome,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6513,1,1.0,0.0,1.0,0.0,1.0,1.0,0.982,1.0,Motor milestone achievement at 14 months |
| HLT011-000268,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8317,1,0.4,1.0,1.0,1.0,1.0,0.2,0.99,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000269,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8619,1,1.0,1.0,1.0,0.0,1.0,0.7,0.934,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000270,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9592,1,1.0,1.0,1.0,1.0,1.0,0.7,0.367,1.0,Enzyme activity normalization at 12 months |
| HLT011-000271,MPS II (Hunter),Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5671,1,0.0,0.0,1.0,1.0,1.0,1.0,0.955,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000272,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8459,1,1.0,1.0,0.3,1.0,1.0,1.0,0.863,1.0,6MWT distance change at 48 weeks |
| HLT011-000273,MPS I (Hurler),Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6163,1,0.0,0.0,1.0,1.0,1.0,0.5,0.91,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000274,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8853,1,0.0,1.0,1.0,1.0,1.0,0.7,0.846,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000275,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.974,1.0,TKV change from baseline at 36 months |
| HLT011-000276,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7616,1,1.0,0.0,1.0,1.0,1.0,1.0,0.948,1.0,Aortic root z-score change at 36 months |
| HLT011-000277,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5896,1,1.0,0.0,0.0,1.0,1.0,0.7,0.975,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000278,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7115,1,0.0,1.0,1.0,0.0,1.0,1.0,0.944,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000279,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.405,0,0.0,0.0,1.0,0.0,1.0,0.2,0.865,1.0,6MWT distance change at 48 weeks |
| HLT011-000280,Li-Fraumeni Syndrome,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7478,1,1.0,0.0,1.0,1.0,1.0,1.0,0.859,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000281,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6177,1,1.0,0.0,0.3,1.0,1.0,0.5,0.934,1.0,Enzyme activity normalization at 12 months |
| HLT011-000282,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.764,1,1.0,1.0,0.0,1.0,1.0,1.0,0.978,1.0,6MWT distance change at 48 weeks |
| HLT011-000283,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.853,1.0,Motor milestone achievement at 14 months |
| HLT011-000284,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6092,1,1.0,1.0,0.0,0.0,1.0,0.2,0.896,1.0,Motor milestone achievement at 14 months |
| HLT011-000285,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8698,1,0.0,1.0,1.0,1.0,1.0,0.7,0.888,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000286,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6596,1,1.0,0.0,1.0,1.0,0.0,0.7,0.97,1.0,Aortic root z-score change at 36 months |
| HLT011-000287,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7705,1,1.0,0.0,1.0,1.0,1.0,0.2,0.93,1.0,Enzyme activity normalization at 12 months |
| HLT011-000288,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7575,1,1.0,1.0,1.0,0.0,0.0,0.7,0.918,1.0,Aortic root z-score change at 36 months |
| HLT011-000289,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7736,1,0.0,1.0,1.0,1.0,1.0,0.5,0.69,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000290,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9052,1,0.4,1.0,1.0,1.0,1.0,1.0,0.906,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000291,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5407,0,1.0,0.0,0.3,1.0,1.0,0.2,0.929,1.0,MLMT performance score improvement at 1 year |
| HLT011-000292,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6248,1,1.0,0.0,1.0,0.0,1.0,1.0,0.315,1.0,Enzyme activity normalization at 12 months |
| HLT011-000293,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7684,1,1.0,0.0,1.0,1.0,1.0,1.0,0.977,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000294,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8127,1,1.0,1.0,1.0,0.0,1.0,0.7,0.856,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000295,Usher Syndrome Type 1,Rare auditory,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5929,0,1.0,0.0,1.0,0.0,1.0,1.0,0.732,1.0,MLMT performance score improvement at 1 year |
| HLT011-000296,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7123,1,1.0,0.0,1.0,1.0,1.0,1.0,0.96,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000297,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.446,0,1.0,0.0,1.0,0.0,0.0,0.2,0.937,1.0,Aortic root z-score change at 36 months |
| HLT011-000298,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5362,0,1.0,0.0,0.0,1.0,1.0,0.7,0.629,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000299,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8713,1,1.0,1.0,1.0,0.0,1.0,0.7,0.955,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000300,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.841,1.0,TKV change from baseline at 36 months |
| HLT011-000301,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6977,1,1.0,1.0,0.0,1.0,1.0,0.2,0.947,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000302,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7668,1,0.4,1.0,1.0,0.0,1.0,1.0,0.922,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000303,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.841,1,0.0,1.0,1.0,1.0,1.0,1.0,0.628,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000304,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6271,1,0.0,1.0,0.0,1.0,1.0,0.2,0.889,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000305,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5081,0,1.0,0.0,1.0,0.0,1.0,1.0,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000306,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5634,1,1.0,0.0,1.0,0.0,1.0,1.0,0.371,1.0,Aortic root z-score change at 36 months |
| HLT011-000307,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7663,1,1.0,0.0,1.0,1.0,1.0,1.0,0.951,1.0,Aortic root z-score change at 36 months |
| HLT011-000308,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8102,1,1.0,1.0,0.3,1.0,1.0,1.0,0.923,1.0,TKV change from baseline at 36 months |
| HLT011-000309,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.7116,1,0.0,1.0,1.0,0.0,1.0,0.2,0.979,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000310,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9205,1,0.4,1.0,1.0,1.0,1.0,1.0,0.983,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000311,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6247,1,1.0,0.0,1.0,0.0,1.0,1.0,0.977,1.0,Aortic root z-score change at 36 months |
| HLT011-000312,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4702,0,1.0,0.0,1.0,0.0,0.0,0.7,0.977,1.0,Aortic root z-score change at 36 months |
| HLT011-000313,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8172,1,1.0,1.0,0.0,1.0,1.0,1.0,0.76,1.0,Motor milestone achievement at 14 months |
| HLT011-000314,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.818,1.0,Aortic root z-score change at 36 months |
| HLT011-000315,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.906,1,1.0,1.0,1.0,1.0,0.0,1.0,0.967,1.0,Aortic root z-score change at 36 months |
| HLT011-000316,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3789,0,0.0,0.0,0.0,1.0,1.0,0.5,0.729,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000317,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7091,1,1.0,0.0,1.0,1.0,1.0,0.5,0.032,1.0,Aortic root z-score change at 36 months |
| HLT011-000318,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5293,0,0.0,1.0,0.3,0.0,1.0,1.0,0.84,1.0,TKV change from baseline at 36 months |
| HLT011-000319,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9814,1,1.0,1.0,1.0,1.0,1.0,0.7,0.928,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000320,Down Syndrome,Chromosomal,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2786,0,0.0,0.0,0.3,0.0,1.0,0.2,0.636,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000321,Down Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5169,0,0.4,0.0,1.0,0.0,1.0,0.5,0.898,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000322,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6854,1,1.0,0.0,1.0,1.0,1.0,0.2,0.971,1.0,Aortic root z-score change at 36 months |
| HLT011-000323,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5936,1,1.0,0.0,0.0,1.0,1.0,1.0,0.97,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000324,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6474,1,0.4,1.0,0.0,1.0,1.0,0.7,0.058,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000325,Pendred Syndrome,Rare auditory,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5474,0,0.0,0.0,1.0,1.0,1.0,0.7,0.47,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000326,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9775,1,1.0,1.0,1.0,1.0,1.0,0.5,0.715,1.0,TKV change from baseline at 36 months |
| HLT011-000327,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.9925,1,1.0,1.0,1.0,1.0,1.0,0.2,0.974,1.0,MLMT performance score improvement at 1 year |
| HLT011-000328,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.755,1,0.4,1.0,0.3,1.0,1.0,0.7,0.888,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000329,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6681,1,1.0,0.0,1.0,1.0,0.0,0.7,0.674,1.0,TKV change from baseline at 36 months |
| HLT011-000330,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9188,1,1.0,1.0,1.0,1.0,0.0,1.0,0.768,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000331,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7553,1,0.4,1.0,1.0,0.0,1.0,1.0,0.0,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000332,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7654,1,0.4,1.0,0.3,1.0,1.0,0.5,0.961,1.0,6MWT distance change at 48 weeks |
| HLT011-000333,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9183,1,1.0,1.0,1.0,0.0,1.0,1.0,0.949,1.0,TKV change from baseline at 36 months |
| HLT011-000334,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6891,1,1.0,0.0,1.0,1.0,1.0,1.0,0.975,1.0,MLMT performance score improvement at 1 year |
| HLT011-000335,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.945,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000336,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5777,1,0.0,0.0,1.0,1.0,1.0,1.0,0.713,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000337,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7588,1,1.0,1.0,0.3,1.0,0.0,1.0,0.851,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000338,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,1.0,1,1.0,1.0,1.0,1.0,1.0,0.2,0.947,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000339,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5276,1,1.0,0.0,1.0,0.0,1.0,0.2,0.906,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000340,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6812,1,1.0,0.0,1.0,1.0,1.0,0.5,0.975,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000341,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7162,1,0.4,1.0,0.3,1.0,1.0,1.0,0.0,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000342,Turner Syndrome,Chromosomal,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7527,1,1.0,0.0,1.0,1.0,1.0,0.7,0.965,1.0,Enzyme activity normalization at 12 months |
| HLT011-000343,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7478,1,1.0,0.0,1.0,1.0,1.0,1.0,0.65,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000344,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4473,0,0.4,0.0,1.0,0.0,1.0,0.7,0.721,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000345,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8904,1,1.0,1.0,1.0,1.0,1.0,0.2,0.723,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000346,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6496,1,1.0,1.0,0.3,0.0,1.0,0.2,0.0,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000347,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.3838,0,1.0,0.0,0.0,0.0,1.0,1.0,0.0,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000348,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.791,1.0,Motor milestone achievement at 14 months |
| HLT011-000349,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4916,0,0.0,0.0,1.0,1.0,0.0,0.7,0.981,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000350,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.962,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000351,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.615,1,1.0,0.0,1.0,0.0,1.0,1.0,0.97,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000352,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9782,1,1.0,1.0,1.0,1.0,1.0,1.0,0.978,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000353,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5326,0,0.4,1.0,0.0,0.0,1.0,0.7,0.668,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000354,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.9136,1,0.4,1.0,1.0,1.0,1.0,1.0,0.922,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000355,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3815,0,0.4,0.0,0.0,1.0,0.0,1.0,0.896,1.0,Aortic root z-score change at 36 months |
| HLT011-000356,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6323,1,0.0,1.0,0.0,1.0,1.0,1.0,0.804,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000357,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7385,1,1.0,0.0,1.0,1.0,1.0,0.7,0.496,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000358,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5897,1,1.0,0.0,1.0,0.0,1.0,1.0,0.923,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000359,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5343,0,0.0,1.0,0.0,1.0,0.0,0.7,0.994,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000360,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.865,1,0.0,1.0,1.0,1.0,1.0,1.0,0.671,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000361,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.711,1,1.0,0.0,1.0,1.0,1.0,1.0,0.998,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000362,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.854,1,1.0,1.0,1.0,1.0,0.0,1.0,0.948,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000363,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6729,1,0.4,1.0,1.0,0.0,1.0,0.7,0.83,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000364,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4332,0,0.0,0.0,0.3,1.0,1.0,1.0,0.784,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000365,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.796,1.0,TKV change from baseline at 36 months |
| HLT011-000366,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6373,1,0.4,0.0,1.0,1.0,1.0,0.7,0.93,1.0,Aortic root z-score change at 36 months |
| HLT011-000367,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8612,1,1.0,1.0,0.3,1.0,1.0,1.0,0.841,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000368,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8676,1,0.0,1.0,1.0,1.0,1.0,0.7,0.898,1.0,TKV change from baseline at 36 months |
| HLT011-000369,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7101,1,1.0,0.0,1.0,1.0,1.0,0.2,0.807,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000370,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7772,1,1.0,0.0,1.0,1.0,1.0,0.7,0.955,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000371,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7521,1,1.0,0.0,1.0,1.0,1.0,1.0,0.977,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000372,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5668,0,0.0,1.0,1.0,0.0,0.0,1.0,0.731,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000373,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8408,1,1.0,1.0,0.3,1.0,1.0,1.0,0.953,1.0,Aortic root z-score change at 36 months |
| HLT011-000374,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.4826,0,1.0,0.0,0.3,1.0,0.0,1.0,0.976,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000375,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7973,1,0.4,1.0,1.0,1.0,0.0,1.0,0.935,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000376,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.79,1,0.4,1.0,0.3,1.0,1.0,1.0,0.864,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000377,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4316,0,0.0,0.0,0.0,1.0,1.0,0.7,0.733,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000378,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5613,1,1.0,0.0,0.3,1.0,1.0,1.0,0.758,1.0,Aortic root z-score change at 36 months |
| HLT011-000379,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5614,1,1.0,0.0,0.0,1.0,1.0,1.0,0.987,1.0,Aortic root z-score change at 36 months |
| HLT011-000380,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8602,1,0.4,1.0,1.0,1.0,1.0,0.7,0.78,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000381,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5736,1,0.0,1.0,0.3,0.0,1.0,1.0,0.978,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000382,Down Syndrome,Chromosomal,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.4822,0,1.0,0.0,0.0,1.0,1.0,0.7,0.971,1.0,6MWT distance change at 48 weeks |
| HLT011-000383,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.992,1,1.0,1.0,1.0,1.0,1.0,0.2,0.871,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000384,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5781,1,1.0,0.0,1.0,0.0,1.0,1.0,0.874,1.0,Aortic root z-score change at 36 months |
| HLT011-000385,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5712,1,1.0,0.0,1.0,0.0,1.0,0.7,0.974,1.0,Aortic root z-score change at 36 months |
| HLT011-000386,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5659,1,1.0,0.0,1.0,0.0,1.0,1.0,0.932,1.0,Aortic root z-score change at 36 months |
| HLT011-000387,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7957,1,1.0,1.0,1.0,0.0,1.0,0.2,0.911,1.0,TKV change from baseline at 36 months |
| HLT011-000388,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5942,0,1.0,0.0,1.0,0.0,1.0,0.7,0.778,1.0,MLMT performance score improvement at 1 year |
| HLT011-000389,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8511,1,1.0,1.0,0.3,1.0,1.0,1.0,0.891,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000390,Li-Fraumeni Syndrome,Rare oncological,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.496,0,1.0,0.0,1.0,0.0,0.0,0.7,0.904,1.0,MLMT performance score improvement at 1 year |
| HLT011-000391,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.3765,0,0.0,0.0,1.0,0.0,0.0,1.0,0.562,1.0,TKV change from baseline at 36 months |
| HLT011-000392,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8192,1,1.0,1.0,1.0,0.0,1.0,0.7,0.716,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000393,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7757,1,1.0,1.0,1.0,0.0,1.0,1.0,0.0,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000394,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4909,0,0.4,1.0,0.0,0.0,0.0,1.0,0.962,1.0,TKV change from baseline at 36 months |
| HLT011-000395,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8066,1,1.0,1.0,1.0,0.0,1.0,0.2,0.951,1.0,Enzyme activity normalization at 12 months |
| HLT011-000396,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7498,1,1.0,0.0,1.0,1.0,1.0,0.2,0.945,1.0,Aortic root z-score change at 36 months |
| HLT011-000397,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4061,0,1.0,0.0,0.0,0.0,1.0,1.0,0.819,1.0,MLMT performance score improvement at 1 year |
| HLT011-000398,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9745,1,1.0,1.0,1.0,1.0,1.0,0.5,0.948,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000399,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.533,0,1.0,0.0,0.0,1.0,1.0,0.5,0.753,1.0,Aortic root z-score change at 36 months |
| HLT011-000400,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5529,1,1.0,0.0,0.3,1.0,0.0,1.0,0.481,1.0,Aortic root z-score change at 36 months |
| HLT011-000401,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8923,1,1.0,1.0,0.3,1.0,1.0,1.0,0.0,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000402,Down Syndrome,Chromosomal,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7533,1,1.0,0.0,1.0,1.0,1.0,1.0,0.95,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000403,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6749,1,0.4,1.0,1.0,0.0,0.0,1.0,0.42,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000404,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9593,1,1.0,1.0,1.0,1.0,1.0,0.7,0.888,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000405,Methylmalonic Acidemia,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7252,1,1.0,0.0,1.0,1.0,1.0,0.2,0.908,1.0,Enzyme activity normalization at 12 months |
| HLT011-000406,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4811,0,1.0,0.0,0.3,1.0,0.0,0.2,0.788,1.0,Aortic root z-score change at 36 months |
| HLT011-000407,Rett Syndrome,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4728,0,0.0,0.0,1.0,1.0,0.0,1.0,0.927,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000408,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.3969,0,1.0,0.0,0.3,0.0,1.0,0.7,0.887,1.0,MLMT performance score improvement at 1 year |
| HLT011-000409,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7715,1,1.0,1.0,1.0,0.0,1.0,0.7,0.855,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000410,Propionic Acidemia,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.2335,0,0.0,0.0,0.0,0.0,1.0,1.0,0.91,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000411,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8626,1,0.4,1.0,1.0,1.0,1.0,0.2,0.778,1.0,TKV change from baseline at 36 months |
| HLT011-000412,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7781,1,0.0,1.0,1.0,1.0,0.0,1.0,0.0,1.0,TKV change from baseline at 36 months |
| HLT011-000413,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8109,1,0.4,1.0,1.0,1.0,0.0,0.2,0.99,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000414,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5378,0,1.0,0.0,1.0,0.0,1.0,0.2,0.955,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000415,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7193,1,0.0,1.0,0.3,1.0,1.0,1.0,0.962,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000416,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9651,1,1.0,1.0,1.0,1.0,1.0,0.5,0.909,1.0,Motor milestone achievement at 14 months |
| HLT011-000417,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8386,1,1.0,1.0,1.0,0.0,1.0,1.0,0.905,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000418,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7526,1,1.0,1.0,1.0,0.0,0.0,1.0,0.902,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000419,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8667,1,1.0,1.0,1.0,0.0,1.0,1.0,0.936,1.0,6MWT distance change at 48 weeks |
| HLT011-000420,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.5511,1,0.4,0.0,0.3,1.0,1.0,1.0,0.972,1.0,6MWT distance change at 48 weeks |
| HLT011-000421,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8618,1,0.4,1.0,1.0,1.0,1.0,0.7,0.936,1.0,TKV change from baseline at 36 months |
| HLT011-000422,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8481,1,0.0,1.0,1.0,1.0,1.0,1.0,0.93,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000423,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.897,1.0,Enzyme activity normalization at 12 months |
| HLT011-000424,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.4259,0,0.4,0.0,0.3,1.0,0.0,1.0,0.973,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000425,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.3969,0,0.0,0.0,0.0,1.0,1.0,1.0,0.805,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000426,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7343,1,1.0,0.0,1.0,1.0,1.0,0.2,0.962,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000427,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6914,1,0.4,1.0,0.0,1.0,1.0,0.7,0.838,1.0,Motor milestone achievement at 14 months |
| HLT011-000428,Li-Fraumeni Syndrome,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.393,0,1.0,0.0,0.0,0.0,1.0,1.0,0.937,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000429,MPS II (Hunter),Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7501,1,1.0,0.0,1.0,1.0,1.0,0.7,0.972,1.0,Enzyme activity normalization at 12 months |
| HLT011-000430,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7049,1,1.0,0.0,1.0,1.0,1.0,1.0,0.349,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000431,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.619,1,0.0,0.0,1.0,1.0,1.0,1.0,0.967,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000432,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8808,1,1.0,1.0,1.0,0.0,1.0,1.0,0.941,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000433,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8874,1,0.4,1.0,1.0,1.0,1.0,0.2,0.946,1.0,TKV change from baseline at 36 months |
| HLT011-000434,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6289,1,1.0,0.0,1.0,1.0,0.0,0.7,0.982,1.0,MLMT performance score improvement at 1 year |
| HLT011-000435,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7013,1,0.0,1.0,0.3,1.0,1.0,0.7,0.655,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000436,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8989,1,0.4,1.0,1.0,1.0,1.0,1.0,0.94,1.0,6MWT distance change at 48 weeks |
| HLT011-000437,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8735,1,1.0,1.0,1.0,0.0,1.0,1.0,0.847,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000438,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7817,1,0.0,1.0,1.0,1.0,1.0,1.0,0.556,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000439,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8991,1,0.4,1.0,1.0,1.0,1.0,1.0,0.973,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000440,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6081,1,0.4,0.0,1.0,1.0,1.0,1.0,0.763,1.0,Aortic root z-score change at 36 months |
| HLT011-000441,Li-Fraumeni Syndrome,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7671,1,1.0,0.0,1.0,1.0,1.0,0.7,0.607,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000442,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.993,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000443,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8359,1,1.0,1.0,0.3,1.0,1.0,1.0,0.917,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000444,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9514,1,1.0,1.0,1.0,1.0,1.0,1.0,0.883,1.0,6MWT distance change at 48 weeks |
| HLT011-000445,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5883,0,0.0,1.0,1.0,0.0,1.0,0.5,0.908,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000446,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8214,1,1.0,1.0,0.3,1.0,1.0,0.7,0.657,1.0,TKV change from baseline at 36 months |
| HLT011-000447,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6594,1,1.0,0.0,1.0,0.0,1.0,0.7,0.961,1.0,Aortic root z-score change at 36 months |
| HLT011-000448,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4266,0,0.0,0.0,1.0,0.0,1.0,1.0,0.778,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000449,MPS I (Hurler),Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6487,1,1.0,0.0,1.0,1.0,0.0,0.7,0.89,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000450,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.4881,0,1.0,0.0,1.0,0.0,0.0,1.0,0.901,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000451,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7399,1,1.0,0.0,1.0,1.0,1.0,0.7,0.964,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000452,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7612,1,1.0,0.0,1.0,1.0,1.0,0.7,0.982,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000453,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8469,1,1.0,1.0,1.0,0.0,1.0,0.7,0.951,1.0,TKV change from baseline at 36 months |
| HLT011-000454,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.974,1.0,6MWT distance change at 48 weeks |
| HLT011-000455,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8827,1,0.4,1.0,1.0,1.0,1.0,1.0,0.779,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000456,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7212,1,1.0,1.0,0.3,0.0,1.0,0.7,0.875,1.0,6MWT distance change at 48 weeks |
| HLT011-000457,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5085,0,1.0,0.0,0.3,0.0,1.0,1.0,0.986,1.0,Aortic root z-score change at 36 months |
| HLT011-000458,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3694,0,1.0,0.0,0.0,0.0,1.0,1.0,0.95,1.0,Aortic root z-score change at 36 months |
| HLT011-000459,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7909,1,1.0,1.0,1.0,0.0,1.0,0.2,0.986,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000460,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4413,0,0.0,0.0,1.0,0.0,1.0,0.2,0.441,1.0,TKV change from baseline at 36 months |
| HLT011-000461,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7491,1,0.4,1.0,0.3,1.0,1.0,0.7,0.896,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000462,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.915,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000463,Down Syndrome,Chromosomal,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.4757,0,0.4,0.0,1.0,0.0,1.0,0.5,0.967,1.0,Enzyme activity normalization at 12 months |
| HLT011-000464,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7917,1,1.0,1.0,1.0,0.0,0.0,1.0,0.933,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000465,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7989,1,0.0,1.0,1.0,1.0,1.0,0.2,0.105,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000466,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8937,1,1.0,1.0,1.0,0.0,1.0,1.0,0.893,1.0,TKV change from baseline at 36 months |
| HLT011-000467,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8954,1,0.4,1.0,1.0,1.0,1.0,1.0,0.874,1.0,TKV change from baseline at 36 months |
| HLT011-000468,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4207,0,0.0,0.0,1.0,0.0,1.0,1.0,0.964,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000469,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9823,1,1.0,1.0,1.0,1.0,1.0,1.0,0.858,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000470,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6016,1,0.0,1.0,0.0,1.0,1.0,0.2,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000471,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5807,1,0.4,0.0,1.0,1.0,1.0,0.2,0.988,1.0,Aortic root z-score change at 36 months |
| HLT011-000472,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7567,1,1.0,0.0,1.0,1.0,1.0,0.7,0.987,1.0,Aortic root z-score change at 36 months |
| HLT011-000473,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9511,1,1.0,1.0,1.0,1.0,1.0,0.2,0.974,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000474,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8768,1,1.0,1.0,1.0,0.0,1.0,1.0,0.745,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000475,Friedreich Ataxia,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5623,1,1.0,0.0,1.0,0.0,1.0,0.7,0.929,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000476,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8419,1,1.0,1.0,1.0,1.0,0.0,1.0,0.751,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000477,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4727,0,1.0,0.0,1.0,0.0,0.0,1.0,0.916,1.0,Aortic root z-score change at 36 months |
| HLT011-000478,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4567,0,0.0,0.0,1.0,0.0,1.0,1.0,0.975,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000479,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.2874,0,0.0,0.0,0.3,0.0,1.0,0.7,0.825,1.0,TKV change from baseline at 36 months |
| HLT011-000480,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7218,1,1.0,0.0,1.0,1.0,1.0,0.5,0.98,1.0,Aortic root z-score change at 36 months |
| HLT011-000481,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.544,0,1.0,0.0,0.0,1.0,1.0,1.0,0.959,1.0,Aortic root z-score change at 36 months |
| HLT011-000482,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9675,1,1.0,1.0,1.0,1.0,1.0,0.2,0.78,1.0,6MWT distance change at 48 weeks |
| HLT011-000483,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7201,1,0.0,1.0,1.0,0.0,1.0,1.0,0.962,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000484,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6143,1,0.0,0.0,1.0,1.0,1.0,1.0,0.814,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000485,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6902,1,0.0,1.0,1.0,0.0,1.0,0.7,0.687,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000486,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.511,0,1.0,0.0,1.0,0.0,0.0,1.0,0.809,1.0,Aortic root z-score change at 36 months |
| HLT011-000487,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6546,1,0.4,0.0,1.0,1.0,1.0,0.7,0.924,1.0,Aortic root z-score change at 36 months |
| HLT011-000488,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8009,1,0.4,1.0,0.3,1.0,1.0,0.5,0.663,1.0,6MWT distance change at 48 weeks |
| HLT011-000489,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8267,1,0.0,1.0,1.0,1.0,1.0,0.5,0.978,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000490,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6864,1,0.0,1.0,1.0,0.0,1.0,1.0,0.855,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000491,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6597,1,0.0,1.0,1.0,0.0,1.0,0.7,0.809,1.0,TKV change from baseline at 36 months |
| HLT011-000492,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6148,1,0.4,1.0,1.0,0.0,0.0,0.7,0.924,1.0,TKV change from baseline at 36 months |
| HLT011-000493,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9532,1,1.0,1.0,1.0,1.0,1.0,1.0,0.686,1.0,TKV change from baseline at 36 months |
| HLT011-000494,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5598,0,0.0,0.0,1.0,1.0,1.0,0.7,0.574,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000495,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9403,1,1.0,1.0,1.0,1.0,1.0,0.2,0.964,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000496,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7247,1,0.4,1.0,1.0,0.0,1.0,0.7,0.293,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000497,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6087,1,1.0,0.0,1.0,0.0,1.0,1.0,0.956,1.0,Aortic root z-score change at 36 months |
| HLT011-000498,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4506,0,0.0,0.0,1.0,0.0,1.0,1.0,0.969,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000499,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5263,0,0.0,0.0,1.0,1.0,0.0,1.0,0.976,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000500,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8288,1,0.0,1.0,1.0,1.0,1.0,0.2,0.62,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000501,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9796,1,1.0,1.0,1.0,1.0,1.0,0.7,0.881,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000502,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6255,1,1.0,0.0,1.0,1.0,0.0,1.0,0.836,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000503,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7416,1,1.0,0.0,1.0,1.0,1.0,0.7,0.931,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000504,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6226,1,0.0,1.0,1.0,0.0,1.0,0.7,0.269,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000505,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6958,1,0.4,1.0,0.0,1.0,1.0,0.7,0.996,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000506,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.535,0,1.0,0.0,0.0,1.0,1.0,0.5,0.906,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000507,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.393,0,0.0,0.0,1.0,0.0,1.0,1.0,0.0,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000508,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6037,1,1.0,0.0,1.0,0.0,1.0,1.0,0.935,1.0,MLMT performance score improvement at 1 year |
| HLT011-000509,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6315,1,0.0,1.0,1.0,0.0,1.0,0.7,0.571,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000510,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8876,1,1.0,1.0,1.0,0.0,1.0,1.0,0.918,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000511,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7287,1,1.0,0.0,1.0,1.0,1.0,0.7,0.942,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000512,Usher Syndrome Type 1,Rare auditory,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3172,0,0.4,0.0,0.0,0.0,1.0,1.0,0.876,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000513,MPS II (Hunter),Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5627,1,0.0,0.0,1.0,1.0,1.0,1.0,0.914,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000514,Friedreich Ataxia,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6081,1,1.0,0.0,1.0,1.0,0.0,0.2,0.965,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000515,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5914,0,0.0,0.0,1.0,1.0,1.0,1.0,0.0,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000516,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7691,1,0.4,1.0,0.0,1.0,1.0,1.0,0.972,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000517,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6041,1,0.4,0.0,1.0,1.0,1.0,0.5,0.979,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000518,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9674,1,1.0,1.0,1.0,1.0,1.0,1.0,0.75,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000519,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8353,1,1.0,1.0,1.0,0.0,1.0,1.0,0.275,1.0,Enzyme activity normalization at 12 months |
| HLT011-000520,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7979,1,1.0,0.0,1.0,1.0,1.0,1.0,0.986,1.0,MLMT performance score improvement at 1 year |
| HLT011-000521,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5935,0,0.0,0.0,1.0,1.0,1.0,0.7,0.933,1.0,Motor milestone achievement at 14 months |
| HLT011-000522,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.9951,1,1.0,1.0,1.0,1.0,1.0,0.2,0.082,1.0,Aortic root z-score change at 36 months |
| HLT011-000523,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8173,1,1.0,1.0,1.0,1.0,0.0,0.2,0.962,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000524,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6089,1,0.0,1.0,0.0,1.0,1.0,1.0,0.911,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000525,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6846,1,1.0,1.0,0.3,0.0,1.0,0.7,0.977,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000526,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.9702,1,1.0,1.0,1.0,1.0,1.0,0.7,0.928,1.0,Aortic root z-score change at 36 months |
| HLT011-000527,Turner Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5847,0,1.0,0.0,0.3,1.0,1.0,0.2,0.981,1.0,MLMT performance score improvement at 1 year |
| HLT011-000528,Glycogen Storage Disease I,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5736,1,1.0,0.0,1.0,1.0,0.0,0.7,0.63,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000529,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9942,1,1.0,1.0,1.0,1.0,1.0,0.5,0.959,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000530,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5861,0,1.0,0.0,0.0,1.0,1.0,1.0,0.928,1.0,Motor milestone achievement at 14 months |
| HLT011-000531,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.695,1,1.0,1.0,1.0,0.0,0.0,0.2,0.862,1.0,TKV change from baseline at 36 months |
| HLT011-000532,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9572,1,1.0,1.0,1.0,1.0,1.0,0.7,0.609,1.0,TKV change from baseline at 36 months |
| HLT011-000533,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6342,1,0.4,0.0,1.0,1.0,1.0,0.7,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-000534,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8594,1,1.0,1.0,1.0,0.0,1.0,0.7,0.949,1.0,TKV change from baseline at 36 months |
| HLT011-000535,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8522,1,1.0,1.0,0.3,1.0,1.0,0.5,0.958,1.0,Enzyme activity normalization at 12 months |
| HLT011-000536,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7872,1,1.0,1.0,1.0,0.0,1.0,0.7,0.992,1.0,6MWT distance change at 48 weeks |
| HLT011-000537,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.9179,1,0.4,1.0,1.0,1.0,1.0,1.0,0.724,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000538,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6458,1,1.0,1.0,0.3,0.0,1.0,0.2,0.963,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000539,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9787,1,1.0,1.0,1.0,1.0,1.0,0.5,0.723,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000540,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5568,0,1.0,0.0,1.0,0.0,1.0,1.0,0.983,1.0,MLMT performance score improvement at 1 year |
| HLT011-000541,Alagille Syndrome,Rare hepatic,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.3208,0,0.0,0.0,0.3,0.0,1.0,1.0,0.915,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000542,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7892,1,0.0,1.0,1.0,1.0,1.0,0.2,0.848,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000543,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6895,1,1.0,0.0,1.0,1.0,1.0,0.7,0.664,1.0,MLMT performance score improvement at 1 year |
| HLT011-000544,Alagille Syndrome,Rare hepatic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4561,0,0.0,0.0,1.0,0.0,1.0,1.0,0.963,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000545,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9808,1,1.0,1.0,1.0,1.0,1.0,1.0,0.973,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000546,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4914,0,1.0,0.0,0.3,0.0,1.0,0.7,0.996,1.0,Aortic root z-score change at 36 months |
| HLT011-000547,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7218,1,1.0,0.0,1.0,1.0,1.0,0.7,0.9,1.0,Aortic root z-score change at 36 months |
| HLT011-000548,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5823,1,1.0,0.0,1.0,0.0,1.0,0.7,0.777,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000549,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6347,1,1.0,0.0,1.0,1.0,0.0,0.5,0.887,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000550,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3273,0,1.0,0.0,0.3,0.0,0.0,1.0,0.53,1.0,Aortic root z-score change at 36 months |
| HLT011-000551,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6217,1,1.0,0.0,0.3,1.0,1.0,1.0,0.919,1.0,Aortic root z-score change at 36 months |
| HLT011-000552,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7401,1,0.4,1.0,1.0,0.0,1.0,1.0,0.871,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000553,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9726,1,1.0,1.0,1.0,1.0,1.0,1.0,0.497,1.0,6MWT distance change at 48 weeks |
| HLT011-000554,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5653,1,1.0,0.0,1.0,0.0,1.0,1.0,0.885,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000555,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5538,1,0.4,0.0,1.0,1.0,1.0,0.7,0.273,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000556,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.647,1.0,Motor milestone achievement at 14 months |
| HLT011-000557,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9354,1,1.0,1.0,0.3,1.0,1.0,1.0,0.964,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000558,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6514,1,0.0,1.0,0.3,1.0,1.0,0.2,0.517,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000559,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6855,1,0.4,1.0,0.0,1.0,1.0,0.7,0.894,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000560,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6394,1,0.0,1.0,0.0,1.0,1.0,1.0,0.933,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000561,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4359,0,0.0,0.0,0.3,1.0,1.0,0.7,0.786,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000562,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8249,1,1.0,1.0,0.3,1.0,1.0,1.0,0.944,1.0,Motor milestone achievement at 14 months |
| HLT011-000563,Usher Syndrome Type 1,Rare auditory,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6021,1,1.0,0.0,1.0,0.0,1.0,0.7,0.944,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000564,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5138,0,0.0,1.0,0.3,0.0,1.0,1.0,0.954,1.0,TKV change from baseline at 36 months |
| HLT011-000565,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7695,1,1.0,1.0,1.0,0.0,0.0,1.0,0.95,1.0,TKV change from baseline at 36 months |
| HLT011-000566,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8988,1,1.0,1.0,1.0,0.0,1.0,0.7,0.975,1.0,TKV change from baseline at 36 months |
| HLT011-000567,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5134,1,0.4,0.0,1.0,0.0,1.0,1.0,0.941,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000568,MPS II (Hunter),Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7396,1,1.0,0.0,1.0,1.0,1.0,1.0,0.989,1.0,Enzyme activity normalization at 12 months |
| HLT011-000569,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7029,1,1.0,1.0,0.3,0.0,1.0,1.0,0.905,1.0,Aortic root z-score change at 36 months |
| HLT011-000570,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6888,1,0.0,1.0,0.3,1.0,1.0,0.2,0.746,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000571,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7116,1,1.0,0.0,1.0,1.0,1.0,1.0,0.989,1.0,Motor milestone achievement at 14 months |
| HLT011-000572,Usher Syndrome Type 1,Rare auditory,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5717,1,1.0,0.0,1.0,1.0,0.0,0.7,0.52,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000573,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7498,1,1.0,0.0,1.0,1.0,1.0,1.0,0.829,1.0,Aortic root z-score change at 36 months |
| HLT011-000574,Epidermolysis Bullosa,Rare dermatological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5264,0,0.0,0.0,1.0,1.0,0.0,0.5,0.7,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000575,Li-Fraumeni Syndrome,Rare oncological,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5642,0,1.0,0.0,1.0,0.0,1.0,0.7,0.382,1.0,MLMT performance score improvement at 1 year |
| HLT011-000576,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7495,1,0.0,1.0,1.0,0.0,1.0,1.0,0.983,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000577,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.3793,0,0.0,0.0,0.0,1.0,1.0,0.2,0.791,1.0,Motor milestone achievement at 14 months |
| HLT011-000578,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4477,0,0.0,0.0,1.0,0.0,1.0,1.0,0.602,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000579,Li-Fraumeni Syndrome,Rare oncological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6275,1,0.4,0.0,1.0,1.0,1.0,0.2,0.806,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000580,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4355,0,0.0,0.0,0.0,1.0,1.0,1.0,0.972,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000581,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4836,0,0.0,1.0,0.0,0.0,1.0,1.0,0.776,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000582,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4489,0,0.0,1.0,0.0,0.0,1.0,1.0,0.987,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000583,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7506,1,0.4,1.0,1.0,0.0,1.0,1.0,0.703,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000584,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6225,1,1.0,0.0,1.0,1.0,0.0,1.0,0.987,1.0,Aortic root z-score change at 36 months |
| HLT011-000585,Down Syndrome,Chromosomal,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.497,0,1.0,0.0,1.0,0.0,0.0,1.0,0.98,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000586,Wiskott-Aldrich Syndrome,Rare immunological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.1777,0,0.0,0.0,0.0,0.0,1.0,0.7,0.697,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000587,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.5699,1,0.4,1.0,0.3,0.0,1.0,1.0,0.947,1.0,6MWT distance change at 48 weeks |
| HLT011-000588,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5827,1,1.0,0.0,0.3,1.0,1.0,1.0,0.613,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000589,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6159,1,1.0,1.0,0.3,0.0,1.0,0.7,0.829,1.0,6MWT distance change at 48 weeks |
| HLT011-000590,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9804,1,1.0,1.0,1.0,1.0,1.0,0.2,0.628,1.0,Enzyme activity normalization at 12 months |
| HLT011-000591,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7241,1,1.0,0.0,1.0,1.0,1.0,0.2,0.931,1.0,MLMT performance score improvement at 1 year |
| HLT011-000592,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5678,1,1.0,0.0,1.0,0.0,1.0,1.0,0.641,1.0,Aortic root z-score change at 36 months |
| HLT011-000593,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.5878,1,1.0,0.0,1.0,0.0,1.0,0.2,0.826,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000594,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4653,0,0.0,0.0,0.3,1.0,1.0,1.0,0.828,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000595,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9085,1,1.0,1.0,1.0,0.0,1.0,0.7,0.967,1.0,Enzyme activity normalization at 12 months |
| HLT011-000596,Friedreich Ataxia,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.4795,0,1.0,0.0,1.0,0.0,0.0,0.2,0.995,1.0,Motor milestone achievement at 14 months |
| HLT011-000597,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6909,1,0.0,1.0,0.3,1.0,1.0,0.7,0.93,1.0,TKV change from baseline at 36 months |
| HLT011-000598,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4702,0,0.0,0.0,1.0,0.0,1.0,1.0,0.856,1.0,TKV change from baseline at 36 months |
| HLT011-000599,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6607,1,1.0,0.0,1.0,1.0,0.0,1.0,0.732,1.0,Aortic root z-score change at 36 months |
| HLT011-000600,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4852,0,0.0,0.0,1.0,0.0,1.0,1.0,0.922,1.0,TKV change from baseline at 36 months |
| HLT011-000601,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7339,1,1.0,0.0,1.0,1.0,1.0,1.0,0.985,1.0,Aortic root z-score change at 36 months |
| HLT011-000602,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6978,1,1.0,0.0,1.0,1.0,1.0,0.2,0.763,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000603,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4121,0,1.0,0.0,0.0,0.0,1.0,1.0,0.614,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000604,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7522,1,0.0,1.0,0.3,1.0,1.0,1.0,0.909,1.0,TKV change from baseline at 36 months |
| HLT011-000605,Prader-Willi Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.4404,0,0.4,0.0,0.0,1.0,1.0,1.0,0.805,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000606,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8365,1,1.0,1.0,1.0,0.0,1.0,1.0,0.973,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000607,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7202,1,1.0,0.0,1.0,1.0,1.0,0.5,0.919,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000608,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9369,1,0.4,1.0,1.0,1.0,1.0,1.0,0.943,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000609,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8414,1,0.4,1.0,1.0,1.0,1.0,1.0,0.966,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000610,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.376,0,0.4,0.0,1.0,0.0,0.0,1.0,0.692,1.0,Aortic root z-score change at 36 months |
| HLT011-000611,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6853,1,0.4,1.0,0.0,1.0,1.0,1.0,0.959,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000612,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5498,0,0.4,1.0,0.3,0.0,1.0,1.0,0.982,1.0,TKV change from baseline at 36 months |
| HLT011-000613,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.2418,0,0.0,0.0,0.3,0.0,1.0,0.2,0.756,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000614,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7064,1,1.0,0.0,1.0,1.0,1.0,0.7,0.863,1.0,Aortic root z-score change at 36 months |
| HLT011-000615,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8518,1,1.0,1.0,0.3,1.0,1.0,1.0,0.927,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000616,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7607,1,0.4,1.0,1.0,0.0,1.0,0.2,0.977,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000617,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9586,1,1.0,1.0,1.0,1.0,1.0,0.2,0.932,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000618,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7309,1,1.0,0.0,1.0,1.0,1.0,1.0,0.42,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000619,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.991,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000620,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7994,1,0.0,1.0,1.0,1.0,1.0,0.5,0.954,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000621,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9448,1,1.0,1.0,1.0,1.0,1.0,0.7,0.472,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000622,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7167,1,0.0,1.0,1.0,0.0,1.0,1.0,0.997,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000623,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.721,1,1.0,0.0,1.0,1.0,1.0,0.7,0.654,1.0,Aortic root z-score change at 36 months |
| HLT011-000624,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5519,1,1.0,0.0,0.0,1.0,1.0,1.0,0.415,1.0,Aortic root z-score change at 36 months |
| HLT011-000625,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7335,1,1.0,0.0,1.0,1.0,1.0,0.7,0.864,1.0,Aortic root z-score change at 36 months |
| HLT011-000626,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.767,1,1.0,0.0,1.0,1.0,1.0,1.0,0.864,1.0,Aortic root z-score change at 36 months |
| HLT011-000627,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7172,1,1.0,1.0,1.0,0.0,0.0,1.0,0.943,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000628,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7373,1,1.0,0.0,1.0,1.0,1.0,0.7,0.989,1.0,Enzyme activity normalization at 12 months |
| HLT011-000629,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5368,0,0.4,1.0,0.0,0.0,1.0,1.0,0.437,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000630,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5335,0,1.0,0.0,1.0,0.0,1.0,1.0,0.0,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000631,Down Syndrome,Chromosomal,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6147,1,1.0,0.0,1.0,0.0,1.0,1.0,0.805,1.0,Enzyme activity normalization at 12 months |
| HLT011-000632,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5533,0,1.0,0.0,1.0,0.0,1.0,0.2,0.86,1.0,MLMT performance score improvement at 1 year |
| HLT011-000633,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7959,1,0.0,1.0,1.0,1.0,1.0,0.7,0.619,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000634,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7338,1,1.0,1.0,1.0,0.0,0.0,0.7,0.955,1.0,Aortic root z-score change at 36 months |
| HLT011-000635,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.769,1,1.0,0.0,1.0,1.0,1.0,0.7,0.904,1.0,Enzyme activity normalization at 12 months |
| HLT011-000636,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7802,1,1.0,1.0,1.0,0.0,1.0,1.0,0.953,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000637,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9339,1,1.0,1.0,1.0,1.0,1.0,0.5,0.96,1.0,TKV change from baseline at 36 months |
| HLT011-000638,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9395,1,1.0,1.0,1.0,1.0,1.0,0.2,0.973,1.0,TKV change from baseline at 36 months |
| HLT011-000639,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.833,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000640,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6251,1,1.0,0.0,0.3,1.0,1.0,1.0,0.738,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000641,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9637,1,1.0,1.0,1.0,1.0,1.0,1.0,0.931,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000642,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6018,1,1.0,0.0,1.0,1.0,0.0,0.7,0.913,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000643,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5767,1,1.0,0.0,1.0,0.0,1.0,0.7,0.176,1.0,Enzyme activity normalization at 12 months |
| HLT011-000644,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9792,1,1.0,1.0,1.0,1.0,1.0,0.5,0.916,1.0,Motor milestone achievement at 14 months |
| HLT011-000645,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6215,1,0.4,0.0,1.0,1.0,1.0,0.2,0.97,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000646,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5892,1,1.0,0.0,1.0,0.0,1.0,1.0,0.361,1.0,Enzyme activity normalization at 12 months |
| HLT011-000647,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8606,1,1.0,1.0,0.3,1.0,1.0,1.0,0.861,1.0,Enzyme activity normalization at 12 months |
| HLT011-000648,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6546,1,0.0,1.0,0.0,1.0,1.0,0.7,0.775,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000649,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8606,1,0.0,1.0,1.0,1.0,1.0,0.7,0.945,1.0,TKV change from baseline at 36 months |
| HLT011-000650,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.649,1,0.0,1.0,0.3,1.0,1.0,0.7,0.986,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000651,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4636,0,1.0,0.0,0.0,1.0,1.0,1.0,0.368,1.0,Aortic root z-score change at 36 months |
| HLT011-000652,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7556,1,1.0,0.0,1.0,1.0,1.0,1.0,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000653,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8578,1,0.4,1.0,1.0,1.0,1.0,0.5,0.949,1.0,TKV change from baseline at 36 months |
| HLT011-000654,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7435,1,1.0,1.0,0.0,1.0,1.0,1.0,0.976,1.0,Motor milestone achievement at 14 months |
| HLT011-000655,Li-Fraumeni Syndrome,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.5204,0,0.4,0.0,0.3,1.0,1.0,1.0,0.914,1.0,6MWT distance change at 48 weeks |
| HLT011-000656,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6034,1,1.0,0.0,0.0,1.0,1.0,1.0,0.982,1.0,Motor milestone achievement at 14 months |
| HLT011-000657,Li-Fraumeni Syndrome,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7465,1,1.0,0.0,1.0,1.0,1.0,0.7,0.767,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000658,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7879,1,1.0,1.0,1.0,0.0,1.0,0.2,0.369,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000659,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.9096,1,0.4,1.0,1.0,1.0,1.0,1.0,0.976,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000660,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7312,1,1.0,0.0,1.0,1.0,1.0,1.0,0.674,1.0,Aortic root z-score change at 36 months |
| HLT011-000661,Friedreich Ataxia,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.4791,0,1.0,0.0,0.3,1.0,0.0,1.0,0.986,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000662,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7993,1,1.0,0.0,1.0,1.0,1.0,1.0,0.857,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000663,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9431,1,0.4,1.0,1.0,1.0,1.0,1.0,0.967,1.0,6MWT distance change at 48 weeks |
| HLT011-000664,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.569,1,0.0,0.0,1.0,1.0,1.0,1.0,0.888,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000665,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6274,1,1.0,0.0,0.3,1.0,1.0,1.0,0.941,1.0,Motor milestone achievement at 14 months |
| HLT011-000666,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7554,1,1.0,1.0,1.0,0.0,0.0,1.0,0.737,1.0,TKV change from baseline at 36 months |
| HLT011-000667,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.929,1.0,MLMT performance score improvement at 1 year |
| HLT011-000668,Propionic Acidemia,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7344,1,1.0,0.0,1.0,1.0,1.0,0.2,0.911,1.0,Enzyme activity normalization at 12 months |
| HLT011-000669,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8721,1,1.0,1.0,1.0,0.0,1.0,1.0,0.995,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000670,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4663,0,1.0,0.0,0.3,0.0,1.0,0.7,0.973,1.0,Aortic root z-score change at 36 months |
| HLT011-000671,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9958,1,1.0,1.0,1.0,1.0,1.0,0.7,0.919,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000672,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8403,1,0.0,1.0,1.0,1.0,1.0,1.0,0.594,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000673,Methylmalonic Acidemia,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.3151,0,0.0,0.0,0.3,1.0,0.0,0.5,0.727,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000674,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6503,1,0.4,1.0,0.0,1.0,1.0,0.5,0.957,1.0,TKV change from baseline at 36 months |
| HLT011-000675,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7277,1,0.4,1.0,0.0,1.0,1.0,0.2,0.927,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000676,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5901,1,1.0,0.0,0.0,1.0,1.0,1.0,0.976,1.0,Aortic root z-score change at 36 months |
| HLT011-000677,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7567,1,1.0,0.0,1.0,1.0,1.0,1.0,0.967,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000678,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.738,1,0.0,1.0,1.0,0.0,1.0,1.0,0.916,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000679,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8469,1,1.0,1.0,1.0,0.0,1.0,1.0,0.885,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000680,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7505,1,0.4,1.0,1.0,0.0,1.0,0.7,0.881,1.0,Aortic root z-score change at 36 months |
| HLT011-000681,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6211,1,1.0,0.0,0.3,1.0,1.0,1.0,0.973,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000682,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8802,1,1.0,1.0,1.0,1.0,0.0,0.5,0.947,1.0,6MWT distance change at 48 weeks |
| HLT011-000683,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9435,1,1.0,1.0,1.0,1.0,1.0,0.7,0.82,1.0,Enzyme activity normalization at 12 months |
| HLT011-000684,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4579,0,1.0,0.0,0.3,0.0,1.0,1.0,0.983,1.0,Aortic root z-score change at 36 months |
| HLT011-000685,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5561,1,0.0,0.0,1.0,1.0,1.0,0.2,0.684,1.0,TKV change from baseline at 36 months |
| HLT011-000686,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6619,1,1.0,0.0,1.0,1.0,0.0,0.7,0.0,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000687,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.776,1,1.0,0.0,1.0,1.0,1.0,1.0,0.973,1.0,Aortic root z-score change at 36 months |
| HLT011-000688,Turner Syndrome,Chromosomal,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4541,0,0.0,0.0,0.3,1.0,1.0,0.7,0.953,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000689,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.3868,0,0.4,0.0,0.3,1.0,0.0,0.5,0.514,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000690,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7026,1,0.4,1.0,0.0,1.0,1.0,1.0,0.957,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000691,Rett Syndrome,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.3964,0,1.0,0.0,0.0,1.0,0.0,0.5,0.963,1.0,Motor milestone achievement at 14 months |
| HLT011-000692,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.4361,0,1.0,0.0,0.3,0.0,1.0,1.0,0.95,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000693,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.93,1,1.0,1.0,1.0,1.0,1.0,0.2,0.985,1.0,Motor milestone achievement at 14 months |
| HLT011-000694,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.4605,0,1.0,0.0,0.0,1.0,0.0,1.0,0.84,1.0,Motor milestone achievement at 14 months |
| HLT011-000695,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6602,1,1.0,0.0,1.0,1.0,1.0,0.2,0.86,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000696,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8421,1,0.0,1.0,1.0,1.0,1.0,1.0,0.859,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000697,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7975,1,0.4,1.0,0.3,1.0,1.0,1.0,0.922,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000698,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8406,1,0.0,1.0,1.0,1.0,1.0,0.2,0.694,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000699,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9282,1,0.4,1.0,1.0,1.0,1.0,0.7,0.982,1.0,TKV change from baseline at 36 months |
| HLT011-000700,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9611,1,1.0,1.0,1.0,1.0,1.0,0.2,0.732,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000701,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9716,1,1.0,1.0,1.0,1.0,1.0,1.0,0.899,1.0,Motor milestone achievement at 14 months |
| HLT011-000702,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7466,1,1.0,0.0,1.0,1.0,1.0,0.7,0.94,1.0,Motor milestone achievement at 14 months |
| HLT011-000703,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8205,1,0.4,1.0,1.0,1.0,0.0,1.0,0.969,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000704,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6366,1,1.0,0.0,1.0,0.0,1.0,1.0,0.933,1.0,TKV change from baseline at 36 months |
| HLT011-000705,Rett Syndrome,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7114,1,1.0,0.0,1.0,1.0,1.0,0.2,0.911,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000706,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4999,0,0.0,1.0,0.3,0.0,1.0,0.5,0.909,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000707,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9672,1,1.0,1.0,1.0,1.0,1.0,0.5,0.892,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000708,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7017,1,1.0,1.0,0.3,0.0,1.0,0.7,0.905,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000709,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5643,1,1.0,0.0,1.0,0.0,1.0,0.5,0.678,1.0,Aortic root z-score change at 36 months |
| HLT011-000710,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6102,1,0.4,0.0,1.0,1.0,1.0,0.2,0.984,1.0,Aortic root z-score change at 36 months |
| HLT011-000711,Glycogen Storage Disease I,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4334,0,0.0,0.0,1.0,0.0,1.0,0.5,0.86,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000712,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6424,1,0.4,1.0,1.0,0.0,0.0,1.0,0.917,1.0,TKV change from baseline at 36 months |
| HLT011-000713,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7184,1,1.0,0.0,1.0,1.0,1.0,1.0,0.575,1.0,MLMT performance score improvement at 1 year |
| HLT011-000714,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5755,1,0.0,0.0,1.0,1.0,1.0,0.7,0.984,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000715,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6554,1,1.0,1.0,0.0,0.0,1.0,1.0,0.937,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000716,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7365,1,1.0,1.0,1.0,0.0,0.0,0.5,0.944,1.0,6MWT distance change at 48 weeks |
| HLT011-000717,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8744,1,0.4,1.0,1.0,1.0,1.0,1.0,0.951,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000718,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.945,1,1.0,1.0,1.0,1.0,1.0,0.2,0.714,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000719,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7815,1,0.4,1.0,1.0,0.0,1.0,0.5,0.945,1.0,TKV change from baseline at 36 months |
| HLT011-000720,Turner Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6989,1,1.0,0.0,1.0,1.0,1.0,0.7,0.859,1.0,MLMT performance score improvement at 1 year |
| HLT011-000721,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5817,0,0.0,1.0,0.0,1.0,1.0,1.0,0.841,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000722,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5249,0,1.0,0.0,1.0,0.0,1.0,0.7,0.759,1.0,Aortic root z-score change at 36 months |
| HLT011-000723,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.9107,1,0.4,1.0,1.0,1.0,1.0,1.0,0.994,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000724,Turner Syndrome,Chromosomal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5807,1,1.0,0.0,0.3,1.0,1.0,1.0,0.14,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000725,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8741,1,1.0,1.0,1.0,0.0,1.0,0.7,0.988,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000726,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6063,1,0.0,1.0,1.0,0.0,1.0,0.7,0.95,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000727,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9735,1,1.0,1.0,1.0,1.0,1.0,0.2,0.787,1.0,Motor milestone achievement at 14 months |
| HLT011-000728,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4135,0,0.4,0.0,0.3,0.0,1.0,1.0,0.89,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000729,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5327,0,1.0,0.0,1.0,0.0,1.0,0.2,0.951,1.0,Aortic root z-score change at 36 months |
| HLT011-000730,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8056,1,0.0,1.0,1.0,1.0,1.0,0.2,0.967,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000731,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6215,1,1.0,0.0,1.0,1.0,0.0,1.0,0.946,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000732,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6343,1,0.4,0.0,1.0,1.0,1.0,0.7,0.868,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000733,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6177,1,1.0,1.0,0.3,0.0,0.0,0.2,0.895,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000734,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.771,1,1.0,0.0,1.0,1.0,1.0,0.2,0.93,1.0,Aortic root z-score change at 36 months |
| HLT011-000735,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6544,1,0.0,0.0,1.0,1.0,1.0,1.0,0.909,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000736,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8456,1,0.0,1.0,1.0,1.0,1.0,1.0,0.818,1.0,TKV change from baseline at 36 months |
| HLT011-000737,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.902,1,1.0,1.0,1.0,0.0,1.0,1.0,0.978,1.0,6MWT distance change at 48 weeks |
| HLT011-000738,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9948,1,1.0,1.0,1.0,1.0,1.0,1.0,0.921,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000739,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7516,1,1.0,0.0,1.0,1.0,1.0,1.0,0.922,1.0,Aortic root z-score change at 36 months |
| HLT011-000740,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5406,0,1.0,0.0,0.0,1.0,1.0,1.0,0.321,1.0,Aortic root z-score change at 36 months |
| HLT011-000741,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.953,1,1.0,1.0,0.3,1.0,1.0,1.0,0.921,1.0,Enzyme activity normalization at 12 months |
| HLT011-000742,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9151,1,0.4,1.0,1.0,1.0,1.0,0.5,0.985,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000743,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5842,1,1.0,0.0,0.3,1.0,1.0,1.0,0.847,1.0,Enzyme activity normalization at 12 months |
| HLT011-000744,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8877,1,1.0,1.0,0.3,1.0,1.0,1.0,0.954,1.0,Enzyme activity normalization at 12 months |
| HLT011-000745,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8261,1,0.0,1.0,1.0,1.0,1.0,0.7,0.964,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000746,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3685,0,0.0,1.0,0.0,0.0,0.0,0.7,0.841,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000747,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7353,1,1.0,0.0,1.0,1.0,1.0,0.7,0.878,1.0,Motor milestone achievement at 14 months |
| HLT011-000748,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3982,0,1.0,0.0,0.0,0.0,1.0,1.0,0.66,1.0,Aortic root z-score change at 36 months |
| HLT011-000749,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6975,1,1.0,1.0,0.0,1.0,0.0,1.0,0.904,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000750,Usher Syndrome Type 1,Rare auditory,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6575,1,0.4,0.0,1.0,1.0,1.0,0.7,0.98,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000751,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.8449,1,0.0,1.0,1.0,1.0,1.0,1.0,0.404,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000752,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.4473,0,1.0,0.0,0.3,0.0,1.0,0.2,0.976,1.0,Motor milestone achievement at 14 months |
| HLT011-000753,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7542,1,1.0,1.0,0.0,1.0,1.0,0.7,0.855,1.0,Motor milestone achievement at 14 months |
| HLT011-000754,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6935,1,1.0,0.0,1.0,1.0,1.0,0.7,0.96,1.0,Aortic root z-score change at 36 months |
| HLT011-000755,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6635,1,0.0,1.0,1.0,0.0,1.0,0.2,0.91,1.0,TKV change from baseline at 36 months |
| HLT011-000756,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7882,1,1.0,1.0,0.0,1.0,1.0,1.0,0.522,1.0,6MWT distance change at 48 weeks |
| HLT011-000757,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.577,0,0.0,0.0,1.0,1.0,1.0,1.0,0.927,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000758,Down Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3365,0,0.0,0.0,0.0,1.0,1.0,0.5,0.893,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000759,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7352,1,1.0,0.0,1.0,1.0,1.0,1.0,0.914,1.0,Enzyme activity normalization at 12 months |
| HLT011-000760,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7117,1,1.0,0.0,1.0,1.0,1.0,0.5,0.976,1.0,Aortic root z-score change at 36 months |
| HLT011-000761,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.744,1,1.0,1.0,0.3,1.0,0.0,0.7,0.912,1.0,TKV change from baseline at 36 months |
| HLT011-000762,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6675,1,0.4,1.0,1.0,0.0,0.0,1.0,0.97,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000763,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7705,1,1.0,0.0,1.0,1.0,1.0,1.0,0.958,1.0,Enzyme activity normalization at 12 months |
| HLT011-000764,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8964,1,0.4,1.0,1.0,1.0,1.0,0.7,0.883,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000765,Down Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3424,0,0.0,0.0,0.0,1.0,1.0,1.0,0.971,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000766,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9274,1,1.0,1.0,1.0,1.0,1.0,1.0,0.539,1.0,Enzyme activity normalization at 12 months |
| HLT011-000767,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9068,1,0.4,1.0,1.0,1.0,1.0,1.0,0.907,1.0,6MWT distance change at 48 weeks |
| HLT011-000768,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6371,1,0.0,1.0,0.0,1.0,1.0,1.0,0.79,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000769,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8439,1,1.0,1.0,1.0,0.0,1.0,1.0,0.9,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000770,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3544,0,0.0,0.0,0.3,1.0,0.0,0.7,0.989,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000771,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.701,1.0,Motor milestone achievement at 14 months |
| HLT011-000772,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8117,1,0.0,1.0,1.0,1.0,1.0,1.0,0.834,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000773,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7733,1,0.4,1.0,1.0,1.0,0.0,1.0,0.947,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000774,Li-Fraumeni Syndrome,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5815,1,1.0,0.0,1.0,0.0,1.0,1.0,0.095,1.0,TKV change from baseline at 36 months |
| HLT011-000775,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.897,1,1.0,1.0,0.3,1.0,1.0,1.0,0.988,1.0,Enzyme activity normalization at 12 months |
| HLT011-000776,Li-Fraumeni Syndrome,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.2711,0,0.0,0.0,0.0,1.0,0.0,1.0,0.467,1.0,Motor milestone achievement at 14 months |
| HLT011-000777,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5484,0,1.0,0.0,1.0,0.0,1.0,1.0,0.983,1.0,Aortic root z-score change at 36 months |
| HLT011-000778,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9748,1,1.0,1.0,1.0,1.0,1.0,1.0,0.993,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000779,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8124,1,1.0,1.0,1.0,0.0,1.0,0.7,0.705,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000780,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.57,0,0.0,0.0,1.0,1.0,1.0,0.7,0.807,1.0,Motor milestone achievement at 14 months |
| HLT011-000781,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7874,1,1.0,1.0,0.0,1.0,1.0,0.7,0.941,1.0,Motor milestone achievement at 14 months |
| HLT011-000782,Down Syndrome,Chromosomal,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4,0,0.4,0.0,0.3,0.0,1.0,1.0,0.951,1.0,Aortic root z-score change at 36 months |
| HLT011-000783,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5743,1,1.0,0.0,1.0,0.0,1.0,1.0,0.82,1.0,Aortic root z-score change at 36 months |
| HLT011-000784,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3568,0,0.0,0.0,0.0,1.0,1.0,0.7,0.822,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000785,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7463,1,1.0,0.0,1.0,1.0,1.0,0.7,0.796,1.0,Aortic root z-score change at 36 months |
| HLT011-000786,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6114,1,1.0,0.0,1.0,0.0,1.0,1.0,0.99,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000787,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7257,1,1.0,0.0,1.0,1.0,1.0,0.5,0.753,1.0,MLMT performance score improvement at 1 year |
| HLT011-000788,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3845,0,0.0,0.0,0.0,1.0,1.0,1.0,0.63,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000789,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6176,1,1.0,0.0,1.0,0.0,1.0,1.0,0.0,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000790,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8274,1,0.4,1.0,0.3,1.0,1.0,1.0,0.853,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000791,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6997,1,0.4,1.0,0.0,1.0,1.0,1.0,0.881,1.0,6MWT distance change at 48 weeks |
| HLT011-000792,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6985,1,1.0,0.0,1.0,1.0,1.0,0.7,0.961,1.0,Aortic root z-score change at 36 months |
| HLT011-000793,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7127,1,1.0,0.0,1.0,1.0,1.0,0.2,0.755,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000794,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7037,1,1.0,0.0,1.0,1.0,1.0,0.5,0.876,1.0,Enzyme activity normalization at 12 months |
| HLT011-000795,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7793,1,0.4,1.0,0.3,1.0,1.0,1.0,0.912,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000796,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7367,1,1.0,0.0,1.0,1.0,1.0,0.7,0.401,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000797,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7093,1,1.0,1.0,1.0,0.0,0.0,0.5,0.931,1.0,Enzyme activity normalization at 12 months |
| HLT011-000798,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9329,1,0.4,1.0,1.0,1.0,1.0,1.0,0.776,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000799,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9027,1,0.4,1.0,1.0,1.0,1.0,0.2,0.667,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000800,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9366,1,0.4,1.0,1.0,1.0,1.0,0.7,0.851,1.0,TKV change from baseline at 36 months |
| HLT011-000801,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7895,1,0.4,1.0,1.0,1.0,0.0,0.2,0.855,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000802,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6719,1,1.0,0.0,1.0,1.0,1.0,0.7,0.676,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000803,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.3555,0,0.0,0.0,0.0,1.0,1.0,0.2,0.902,1.0,6MWT distance change at 48 weeks |
| HLT011-000804,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.2116,0,0.0,0.0,0.0,0.0,1.0,0.7,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000805,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9683,1,1.0,1.0,1.0,1.0,1.0,0.7,0.913,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000806,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5325,0,0.4,1.0,0.0,0.0,1.0,0.5,0.916,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000807,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8445,1,0.4,1.0,1.0,1.0,0.0,0.5,0.936,1.0,TKV change from baseline at 36 months |
| HLT011-000808,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9018,1,0.4,1.0,1.0,1.0,1.0,1.0,0.895,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000809,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6977,1,0.0,1.0,1.0,0.0,1.0,1.0,0.511,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000810,Alagille Syndrome,Rare hepatic,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.4516,0,1.0,0.0,0.3,0.0,1.0,1.0,0.951,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000811,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6306,1,1.0,1.0,0.0,0.0,1.0,0.7,0.677,1.0,Enzyme activity normalization at 12 months |
| HLT011-000812,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8563,1,1.0,1.0,0.3,1.0,1.0,1.0,0.871,1.0,Enzyme activity normalization at 12 months |
| HLT011-000813,Alagille Syndrome,Rare hepatic,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7819,1,1.0,0.0,1.0,1.0,1.0,1.0,0.899,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000814,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.9561,1,0.4,1.0,1.0,1.0,1.0,1.0,0.889,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000815,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9064,1,0.4,1.0,1.0,1.0,1.0,0.7,0.969,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000816,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.3605,0,0.0,0.0,0.0,1.0,1.0,1.0,0.835,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000817,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7258,1,1.0,0.0,1.0,1.0,1.0,0.5,0.948,1.0,TKV change from baseline at 36 months |
| HLT011-000818,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7963,1,0.4,1.0,0.3,1.0,1.0,1.0,0.936,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000819,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6034,1,0.0,0.0,1.0,1.0,1.0,1.0,0.9,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000820,Friedreich Ataxia,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.2915,0,1.0,0.0,0.0,0.0,0.0,0.2,0.897,1.0,Motor milestone achievement at 14 months |
| HLT011-000821,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6437,1,1.0,0.0,0.3,1.0,1.0,1.0,0.924,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000822,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6451,1,0.0,1.0,0.0,1.0,1.0,0.7,0.947,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000823,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8512,1,1.0,1.0,1.0,1.0,0.0,0.2,0.796,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000824,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9168,1,0.4,1.0,1.0,1.0,1.0,1.0,0.89,1.0,TKV change from baseline at 36 months |
| HLT011-000825,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6409,1,1.0,0.0,1.0,1.0,1.0,0.5,0.962,1.0,MLMT performance score improvement at 1 year |
| HLT011-000826,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.961,1.0,Enzyme activity normalization at 12 months |
| HLT011-000827,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7469,1,1.0,1.0,0.0,1.0,1.0,1.0,0.955,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000828,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9988,1,1.0,1.0,1.0,1.0,1.0,0.7,0.908,1.0,Motor milestone achievement at 14 months |
| HLT011-000829,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6232,1,1.0,0.0,0.3,1.0,1.0,1.0,0.962,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000830,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9313,1,1.0,1.0,1.0,1.0,1.0,0.2,0.939,1.0,Motor milestone achievement at 14 months |
| HLT011-000831,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4217,0,0.0,0.0,0.0,1.0,1.0,1.0,0.354,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000832,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.9235,1,1.0,1.0,1.0,1.0,1.0,0.2,0.0,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000833,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5268,0,1.0,0.0,0.3,0.0,1.0,1.0,0.983,1.0,Aortic root z-score change at 36 months |
| HLT011-000834,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8325,1,0.0,1.0,1.0,1.0,1.0,1.0,0.418,1.0,TKV change from baseline at 36 months |
| HLT011-000835,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.941,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000836,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8839,1,0.0,1.0,1.0,1.0,1.0,0.7,0.913,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000837,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.532,1,1.0,0.0,0.0,1.0,1.0,0.7,0.67,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000838,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5589,1,1.0,0.0,0.3,1.0,1.0,0.5,0.965,1.0,Aortic root z-score change at 36 months |
| HLT011-000839,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5618,1,1.0,0.0,1.0,0.0,1.0,0.2,0.903,1.0,Aortic root z-score change at 36 months |
| HLT011-000840,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4883,0,0.0,1.0,0.0,0.0,1.0,1.0,0.647,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000841,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.957,1,1.0,1.0,1.0,1.0,1.0,1.0,0.931,1.0,Enzyme activity normalization at 12 months |
| HLT011-000842,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8408,1,1.0,1.0,1.0,0.0,1.0,1.0,0.95,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000843,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8274,1,0.0,1.0,1.0,1.0,1.0,1.0,0.989,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000844,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7769,1,1.0,0.0,1.0,1.0,1.0,1.0,0.972,1.0,Aortic root z-score change at 36 months |
| HLT011-000845,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8987,1,0.4,1.0,1.0,1.0,1.0,0.7,0.989,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000846,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.588,1,1.0,0.0,1.0,0.0,1.0,1.0,0.882,1.0,Enzyme activity normalization at 12 months |
| HLT011-000847,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6561,1,0.4,0.0,1.0,1.0,1.0,1.0,0.977,1.0,Aortic root z-score change at 36 months |
| HLT011-000848,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5844,1,1.0,1.0,0.3,0.0,0.0,0.2,0.992,1.0,TKV change from baseline at 36 months |
| HLT011-000849,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7663,1,0.4,1.0,1.0,0.0,1.0,0.7,0.994,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000850,Prader-Willi Syndrome,Chromosomal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.4759,0,0.4,0.0,0.3,1.0,1.0,0.7,0.973,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000851,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8789,1,1.0,1.0,1.0,0.0,1.0,1.0,0.858,1.0,6MWT distance change at 48 weeks |
| HLT011-000852,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7487,1,1.0,1.0,0.0,1.0,1.0,0.2,0.769,1.0,6MWT distance change at 48 weeks |
| HLT011-000853,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5764,0,1.0,0.0,1.0,1.0,0.0,0.7,0.363,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000854,Down Syndrome,Chromosomal,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7625,1,1.0,0.0,1.0,1.0,1.0,1.0,0.954,1.0,Motor milestone achievement at 14 months |
| HLT011-000855,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6399,1,1.0,1.0,0.0,1.0,0.0,1.0,0.527,1.0,6MWT distance change at 48 weeks |
| HLT011-000856,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4823,0,1.0,0.0,0.3,1.0,0.0,0.2,0.915,1.0,Aortic root z-score change at 36 months |
| HLT011-000857,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9305,1,0.4,1.0,1.0,1.0,1.0,1.0,0.947,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000858,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5222,0,1.0,0.0,0.0,1.0,1.0,0.2,0.765,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000859,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5924,1,1.0,0.0,1.0,0.0,1.0,0.5,0.78,1.0,Aortic root z-score change at 36 months |
| HLT011-000860,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6518,1,0.0,1.0,0.3,1.0,1.0,1.0,0.464,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000861,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9147,1,0.4,1.0,1.0,1.0,1.0,1.0,0.824,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000862,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8787,1,0.4,1.0,1.0,1.0,1.0,0.7,0.659,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000863,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8933,1,0.4,1.0,1.0,1.0,1.0,1.0,0.925,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000864,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5644,1,0.0,1.0,0.3,0.0,1.0,0.5,0.943,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000865,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.792,1,0.4,1.0,1.0,0.0,1.0,0.7,0.986,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000866,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6473,1,1.0,0.0,0.3,1.0,1.0,1.0,0.927,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000867,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7385,1,1.0,0.0,1.0,1.0,1.0,1.0,0.727,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000868,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8381,1,1.0,1.0,1.0,0.0,1.0,0.7,0.651,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000869,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.781,1,0.4,1.0,1.0,0.0,1.0,1.0,0.934,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000870,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8535,1,1.0,1.0,1.0,0.0,1.0,1.0,0.996,1.0,6MWT distance change at 48 weeks |
| HLT011-000871,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6715,1,0.4,1.0,0.3,1.0,0.0,1.0,0.919,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000872,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9909,1,1.0,1.0,1.0,1.0,1.0,0.2,0.941,1.0,Enzyme activity normalization at 12 months |
| HLT011-000873,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7261,1,1.0,0.0,1.0,1.0,1.0,0.5,0.929,1.0,Aortic root z-score change at 36 months |
| HLT011-000874,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5763,1,1.0,0.0,1.0,0.0,1.0,1.0,0.939,1.0,Aortic root z-score change at 36 months |
| HLT011-000875,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6743,1,0.0,1.0,1.0,0.0,1.0,0.7,0.957,1.0,TKV change from baseline at 36 months |
| HLT011-000876,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5995,1,1.0,0.0,1.0,0.0,1.0,1.0,0.981,1.0,Aortic root z-score change at 36 months |
| HLT011-000877,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6077,1,1.0,0.0,0.3,1.0,1.0,0.7,0.984,1.0,Aortic root z-score change at 36 months |
| HLT011-000878,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3276,0,0.0,0.0,0.3,0.0,1.0,1.0,0.917,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000879,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.2569,0,0.0,0.0,0.0,0.0,1.0,0.7,0.875,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000880,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6286,1,0.4,1.0,0.3,0.0,1.0,1.0,0.907,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000881,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5404,0,1.0,0.0,0.3,1.0,1.0,0.2,0.982,1.0,Aortic root z-score change at 36 months |
| HLT011-000882,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.821,1,0.0,1.0,1.0,1.0,1.0,0.7,0.935,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000883,Down Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.696,1,1.0,0.0,1.0,1.0,1.0,1.0,0.856,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000884,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4797,0,1.0,0.0,0.0,1.0,1.0,0.2,0.768,1.0,Aortic root z-score change at 36 months |
| HLT011-000885,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8283,1,1.0,1.0,0.3,1.0,1.0,1.0,0.965,1.0,6MWT distance change at 48 weeks |
| HLT011-000886,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3887,0,1.0,0.0,0.0,0.0,1.0,1.0,0.573,1.0,Aortic root z-score change at 36 months |
| HLT011-000887,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7209,1,1.0,0.0,1.0,1.0,1.0,0.5,0.97,1.0,Aortic root z-score change at 36 months |
| HLT011-000888,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.665,1,1.0,1.0,0.3,0.0,1.0,0.2,0.904,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000889,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6226,1,1.0,1.0,0.3,0.0,0.0,1.0,0.801,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000890,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7597,1,0.0,1.0,1.0,1.0,0.0,1.0,0.982,1.0,TKV change from baseline at 36 months |
| HLT011-000891,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.2984,0,1.0,0.0,0.0,0.0,0.0,0.7,0.938,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000892,Turner Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6015,1,1.0,0.0,1.0,0.0,1.0,0.7,0.833,1.0,MLMT performance score improvement at 1 year |
| HLT011-000893,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6334,1,1.0,1.0,0.0,1.0,0.0,1.0,0.887,1.0,Motor milestone achievement at 14 months |
| HLT011-000894,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8373,1,1.0,1.0,1.0,0.0,1.0,1.0,0.941,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000895,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6629,1,1.0,0.0,1.0,1.0,1.0,0.5,0.942,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000896,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5283,0,1.0,0.0,0.0,1.0,1.0,0.5,0.948,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000897,Friedreich Ataxia,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.766,1,1.0,0.0,1.0,1.0,1.0,0.7,0.887,1.0,Motor milestone achievement at 14 months |
| HLT011-000898,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7955,1,0.4,1.0,1.0,0.0,1.0,1.0,0.996,1.0,6MWT distance change at 48 weeks |
| HLT011-000899,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7534,1,1.0,1.0,0.3,1.0,0.0,0.7,0.977,1.0,6MWT distance change at 48 weeks |
| HLT011-000900,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6625,1,0.0,1.0,0.3,1.0,1.0,0.2,0.985,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000901,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6506,1,1.0,0.0,1.0,1.0,0.0,0.7,0.933,1.0,Aortic root z-score change at 36 months |
| HLT011-000902,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4456,0,0.0,1.0,0.0,0.0,1.0,0.2,0.781,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000903,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.687,1,0.4,0.0,1.0,1.0,1.0,1.0,0.913,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000904,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.537,0,1.0,0.0,1.0,0.0,1.0,0.2,0.525,1.0,Aortic root z-score change at 36 months |
| HLT011-000905,Turner Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5183,0,0.4,0.0,1.0,0.0,1.0,0.7,0.872,1.0,MLMT performance score improvement at 1 year |
| HLT011-000906,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7184,1,0.0,1.0,1.0,0.0,1.0,1.0,0.936,1.0,TKV change from baseline at 36 months |
| HLT011-000907,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8072,1,0.0,1.0,1.0,1.0,1.0,0.7,0.929,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000908,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7551,1,0.4,1.0,0.3,1.0,1.0,0.2,0.595,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000909,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9941,1,1.0,1.0,1.0,1.0,1.0,0.5,0.772,1.0,Motor milestone achievement at 14 months |
| HLT011-000910,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.99,1,1.0,1.0,1.0,1.0,1.0,0.7,0.966,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000911,Epidermolysis Bullosa,Rare dermatological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6955,1,1.0,0.0,1.0,1.0,1.0,0.5,0.709,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000912,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7074,1,1.0,0.0,1.0,1.0,1.0,0.5,0.783,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000913,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5606,1,1.0,0.0,1.0,0.0,1.0,1.0,0.641,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000914,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6598,1,0.4,0.0,1.0,1.0,1.0,1.0,0.555,1.0,Aortic root z-score change at 36 months |
| HLT011-000915,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3566,0,0.4,0.0,0.3,1.0,0.0,1.0,0.127,1.0,Aortic root z-score change at 36 months |
| HLT011-000916,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4895,0,1.0,0.0,0.3,1.0,0.0,0.7,0.92,1.0,MLMT performance score improvement at 1 year |
| HLT011-000917,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8109,1,1.0,0.0,1.0,1.0,1.0,0.7,0.985,1.0,Enzyme activity normalization at 12 months |
| HLT011-000918,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.723,1,0.4,1.0,0.3,1.0,1.0,1.0,0.81,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000919,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6017,1,0.4,1.0,0.0,0.0,1.0,1.0,0.919,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000920,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8224,1,0.0,1.0,1.0,1.0,1.0,0.5,0.598,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000921,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8709,1,1.0,1.0,0.3,1.0,1.0,1.0,0.951,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000922,Usher Syndrome Type 1,Rare auditory,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4507,0,0.4,0.0,0.0,1.0,1.0,0.7,0.972,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000923,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6528,1,0.4,0.0,1.0,1.0,1.0,1.0,0.914,1.0,Aortic root z-score change at 36 months |
| HLT011-000924,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7113,1,0.0,1.0,1.0,0.0,1.0,0.7,0.323,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000925,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7455,1,0.4,1.0,1.0,0.0,1.0,1.0,0.973,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000926,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.864,1.0,Motor milestone achievement at 14 months |
| HLT011-000927,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8382,1,1.0,1.0,0.3,1.0,1.0,1.0,0.917,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000928,Li-Fraumeni Syndrome,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4349,0,0.0,0.0,1.0,0.0,1.0,1.0,0.558,1.0,TKV change from baseline at 36 months |
| HLT011-000929,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.2217,0,0.0,0.0,0.0,0.0,1.0,0.7,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000930,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.938,1,0.4,1.0,1.0,1.0,1.0,0.7,0.888,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000931,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8458,1,1.0,1.0,1.0,0.0,1.0,1.0,0.939,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000932,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8282,1,1.0,1.0,1.0,0.0,1.0,0.7,0.869,1.0,Aortic root z-score change at 36 months |
| HLT011-000933,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7028,1,1.0,0.0,1.0,1.0,1.0,0.7,0.942,1.0,Aortic root z-score change at 36 months |
| HLT011-000934,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.605,1,1.0,0.0,1.0,1.0,0.0,0.2,0.797,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000935,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8499,1,1.0,1.0,1.0,1.0,0.0,1.0,0.924,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000936,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7612,1,1.0,1.0,1.0,0.0,1.0,0.2,0.018,1.0,6MWT distance change at 48 weeks |
| HLT011-000937,Down Syndrome,Chromosomal,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.4513,0,0.0,0.0,0.3,1.0,1.0,1.0,0.484,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000938,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7093,1,0.4,1.0,0.3,1.0,0.0,1.0,0.992,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000939,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6967,1,1.0,0.0,1.0,1.0,1.0,0.2,0.875,1.0,Aortic root z-score change at 36 months |
| HLT011-000940,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8738,1,1.0,1.0,1.0,0.0,1.0,1.0,0.357,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000941,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.8406,1,0.0,1.0,1.0,1.0,1.0,0.7,0.842,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000942,Pendred Syndrome,Rare auditory,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7256,1,1.0,0.0,1.0,1.0,1.0,0.7,0.739,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000943,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8715,1,1.0,1.0,0.3,1.0,1.0,1.0,0.952,1.0,6MWT distance change at 48 weeks |
| HLT011-000944,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7261,1,1.0,0.0,1.0,1.0,1.0,0.5,0.808,1.0,Aortic root z-score change at 36 months |
| HLT011-000945,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5747,1,1.0,0.0,1.0,0.0,1.0,0.5,0.711,1.0,Aortic root z-score change at 36 months |
| HLT011-000946,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8442,1,1.0,1.0,0.3,1.0,1.0,1.0,0.882,1.0,Motor milestone achievement at 14 months |
| HLT011-000947,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6663,1,1.0,1.0,0.3,0.0,1.0,1.0,0.941,1.0,TKV change from baseline at 36 months |
| HLT011-000948,Li-Fraumeni Syndrome,Rare oncological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7366,1,1.0,0.0,1.0,1.0,1.0,0.2,0.967,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000949,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7293,1,1.0,0.0,1.0,1.0,1.0,0.7,0.985,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-000950,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3362,0,0.0,0.0,1.0,0.0,0.0,1.0,0.531,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000951,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8503,1,0.0,1.0,1.0,1.0,1.0,0.5,0.88,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000952,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9014,1,1.0,1.0,1.0,1.0,0.0,1.0,0.866,1.0,Motor milestone achievement at 14 months |
| HLT011-000953,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6577,1,1.0,1.0,0.0,1.0,0.0,0.5,0.914,1.0,Motor milestone achievement at 14 months |
| HLT011-000954,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9421,1,0.4,1.0,1.0,1.0,1.0,1.0,0.842,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000955,Down Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6411,1,0.0,0.0,1.0,1.0,1.0,1.0,0.79,1.0,TKV change from baseline at 36 months |
| HLT011-000956,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6471,1,0.4,0.0,1.0,1.0,1.0,0.2,0.664,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000957,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9269,1,1.0,1.0,1.0,1.0,0.0,1.0,0.656,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000958,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8801,1,0.0,1.0,1.0,1.0,1.0,1.0,0.873,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000959,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8629,1,0.0,1.0,1.0,1.0,1.0,1.0,0.817,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-000960,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8679,1,0.0,1.0,1.0,1.0,1.0,1.0,0.998,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000961,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5731,1,0.0,1.0,0.3,0.0,1.0,1.0,0.916,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000962,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5541,1,1.0,0.0,1.0,0.0,1.0,0.7,0.969,1.0,Aortic root z-score change at 36 months |
| HLT011-000963,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7583,1,1.0,1.0,1.0,0.0,0.0,0.7,0.975,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000964,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9332,1,1.0,1.0,1.0,1.0,1.0,0.5,0.794,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000965,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6232,1,0.4,0.0,1.0,1.0,1.0,0.7,0.969,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000966,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7564,1,1.0,0.0,1.0,1.0,1.0,0.2,0.976,1.0,Aortic root z-score change at 36 months |
| HLT011-000967,Down Syndrome,Chromosomal,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.6341,1,0.0,0.0,1.0,1.0,1.0,1.0,0.65,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000968,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7411,1,1.0,1.0,1.0,0.0,0.0,1.0,0.976,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000969,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7798,1,0.0,1.0,1.0,1.0,1.0,0.2,0.88,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000970,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8278,1,1.0,1.0,1.0,0.0,1.0,1.0,0.5,1.0,6MWT distance change at 48 weeks |
| HLT011-000971,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9839,1,1.0,1.0,1.0,1.0,1.0,1.0,0.361,1.0,Motor milestone achievement at 14 months |
| HLT011-000972,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.71,1,0.0,1.0,0.3,1.0,1.0,1.0,0.921,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-000973,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.862,1,1.0,1.0,1.0,0.0,1.0,0.5,0.934,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000974,Down Syndrome,Chromosomal,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3613,0,0.0,0.0,0.0,1.0,1.0,0.7,0.472,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000975,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.52,0,1.0,0.0,0.0,1.0,1.0,1.0,0.99,1.0,Motor milestone achievement at 14 months |
| HLT011-000976,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6494,1,0.4,0.0,1.0,1.0,1.0,0.7,0.596,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000977,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6454,1,0.4,0.0,1.0,1.0,1.0,0.7,0.911,1.0,Aortic root z-score change at 36 months |
| HLT011-000978,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.2854,0,0.4,0.0,0.0,0.0,1.0,1.0,0.93,1.0,Aortic root z-score change at 36 months |
| HLT011-000979,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8148,1,0.0,1.0,1.0,1.0,1.0,0.5,0.729,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000980,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6322,1,1.0,1.0,0.0,0.0,1.0,0.7,0.989,1.0,6MWT distance change at 48 weeks |
| HLT011-000981,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7352,1,1.0,0.0,1.0,1.0,1.0,1.0,0.777,1.0,Aortic root z-score change at 36 months |
| HLT011-000982,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7702,1,1.0,0.0,1.0,1.0,1.0,0.2,0.967,1.0,MLMT performance score improvement at 1 year |
| HLT011-000983,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9617,1,1.0,1.0,1.0,1.0,1.0,0.2,0.889,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000984,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3338,0,1.0,0.0,0.3,0.0,0.0,0.7,0.77,1.0,Aortic root z-score change at 36 months |
| HLT011-000985,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6964,1,0.4,1.0,1.0,0.0,1.0,1.0,0.979,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-000986,Down Syndrome,Chromosomal,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2829,0,0.0,0.0,0.3,0.0,1.0,0.7,0.76,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000987,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.3463,0,0.4,0.0,0.3,0.0,1.0,1.0,0.896,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-000988,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7315,1,1.0,0.0,1.0,1.0,1.0,0.7,0.949,1.0,Enzyme activity normalization at 12 months |
| HLT011-000989,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5858,0,1.0,0.0,0.0,1.0,1.0,1.0,0.906,1.0,Motor milestone achievement at 14 months |
| HLT011-000990,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7487,1,1.0,1.0,0.3,1.0,0.0,0.7,0.963,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000991,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5844,0,0.0,0.0,1.0,1.0,1.0,0.7,0.967,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-000992,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6599,1,0.4,0.0,1.0,1.0,1.0,1.0,0.994,1.0,Lumbar spine BMD change at 24 months |
| HLT011-000993,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8613,1,1.0,1.0,1.0,0.0,1.0,0.2,0.723,1.0,Androstenedione normalization at 24 weeks |
| HLT011-000994,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9121,1,1.0,1.0,1.0,1.0,1.0,0.7,0.791,1.0,6MWT distance change at 48 weeks |
| HLT011-000995,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5178,0,0.0,0.0,1.0,1.0,1.0,0.7,0.661,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-000996,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7179,1,1.0,0.0,1.0,1.0,1.0,0.7,0.874,1.0,Aortic root z-score change at 36 months |
| HLT011-000997,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8491,1,0.0,1.0,1.0,1.0,1.0,1.0,0.957,1.0,TKV change from baseline at 36 months |
| HLT011-000998,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9747,1,1.0,1.0,1.0,1.0,1.0,0.2,0.533,1.0,Enzyme activity normalization at 12 months |
| HLT011-000999,Alagille Syndrome,Rare hepatic,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5874,1,0.0,0.0,1.0,1.0,1.0,1.0,0.964,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001000,Friedreich Ataxia,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6773,1,1.0,0.0,1.0,1.0,0.0,1.0,0.892,1.0,Motor milestone achievement at 14 months |
| HLT011-001001,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5986,1,1.0,0.0,0.3,1.0,1.0,0.7,0.835,1.0,Enzyme activity normalization at 12 months |
| HLT011-001002,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6284,1,1.0,0.0,1.0,0.0,1.0,1.0,0.984,1.0,Aortic root z-score change at 36 months |
| HLT011-001003,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.623,1,0.4,1.0,0.3,0.0,1.0,1.0,0.828,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001004,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5983,0,0.4,1.0,0.3,0.0,1.0,1.0,0.838,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001005,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7661,1,1.0,0.0,1.0,1.0,1.0,1.0,0.838,1.0,TKV change from baseline at 36 months |
| HLT011-001006,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7318,1,1.0,0.0,1.0,1.0,1.0,0.5,0.95,1.0,Aortic root z-score change at 36 months |
| HLT011-001007,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6456,1,0.4,0.0,1.0,1.0,1.0,1.0,0.517,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001008,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.517,0,1.0,0.0,0.3,1.0,1.0,0.7,0.75,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001009,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9241,1,1.0,1.0,1.0,1.0,1.0,0.2,0.955,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001010,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.711,1,1.0,0.0,1.0,1.0,1.0,1.0,0.889,1.0,Aortic root z-score change at 36 months |
| HLT011-001011,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8365,1,1.0,1.0,0.3,1.0,1.0,0.7,0.979,1.0,TKV change from baseline at 36 months |
| HLT011-001012,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.84,1,0.0,1.0,1.0,1.0,1.0,1.0,0.922,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001013,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6731,1,1.0,0.0,1.0,1.0,1.0,0.5,0.61,1.0,Aortic root z-score change at 36 months |
| HLT011-001014,Down Syndrome,Chromosomal,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5386,0,1.0,0.0,1.0,0.0,1.0,0.5,0.481,1.0,Enzyme activity normalization at 12 months |
| HLT011-001015,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6735,1,0.0,0.0,1.0,1.0,1.0,1.0,0.993,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001016,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8443,1,0.4,1.0,1.0,1.0,0.0,0.7,0.74,1.0,6MWT distance change at 48 weeks |
| HLT011-001017,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8366,1,0.0,1.0,1.0,1.0,1.0,1.0,0.965,1.0,TKV change from baseline at 36 months |
| HLT011-001018,Usher Syndrome Type 1,Rare auditory,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3637,0,0.0,0.0,0.0,1.0,1.0,1.0,0.94,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001019,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7845,1,0.0,1.0,1.0,1.0,1.0,1.0,0.726,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001020,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4827,0,0.4,0.0,1.0,0.0,1.0,1.0,0.208,1.0,Aortic root z-score change at 36 months |
| HLT011-001021,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6901,1,1.0,0.0,1.0,1.0,1.0,1.0,0.0,1.0,Enzyme activity normalization at 12 months |
| HLT011-001022,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.8955,1,1.0,1.0,1.0,1.0,0.0,1.0,0.933,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001023,Alagille Syndrome,Rare hepatic,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.4149,0,1.0,0.0,0.0,1.0,0.0,0.7,0.991,1.0,Motor milestone achievement at 14 months |
| HLT011-001024,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6773,1,0.4,1.0,0.0,1.0,1.0,0.2,0.828,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001025,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6865,1,0.0,1.0,1.0,0.0,1.0,0.5,0.85,1.0,TKV change from baseline at 36 months |
| HLT011-001026,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.987,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001027,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7069,1,1.0,0.0,1.0,1.0,1.0,1.0,0.885,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001028,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8783,1,1.0,1.0,0.3,1.0,1.0,1.0,0.757,1.0,Aortic root z-score change at 36 months |
| HLT011-001029,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9635,1,1.0,1.0,1.0,1.0,1.0,0.7,0.983,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001030,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7531,1,1.0,0.0,1.0,1.0,1.0,0.7,0.871,1.0,Aortic root z-score change at 36 months |
| HLT011-001031,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8143,1,0.0,1.0,1.0,1.0,1.0,0.2,0.887,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001032,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.803,1,0.0,1.0,1.0,1.0,1.0,1.0,0.811,1.0,TKV change from baseline at 36 months |
| HLT011-001033,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8386,1,0.0,1.0,1.0,1.0,1.0,1.0,0.937,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001034,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7531,1,1.0,0.0,1.0,1.0,1.0,0.7,0.889,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001035,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5931,1,1.0,0.0,0.3,1.0,1.0,0.7,0.976,1.0,Aortic root z-score change at 36 months |
| HLT011-001036,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.892,1,1.0,1.0,1.0,0.0,1.0,1.0,0.868,1.0,TKV change from baseline at 36 months |
| HLT011-001037,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6199,1,0.0,0.0,1.0,1.0,1.0,1.0,0.654,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001038,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5909,1,1.0,0.0,1.0,0.0,1.0,1.0,0.952,1.0,Aortic root z-score change at 36 months |
| HLT011-001039,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.747,1,1.0,0.0,1.0,1.0,1.0,1.0,0.151,1.0,Aortic root z-score change at 36 months |
| HLT011-001040,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9254,1,0.4,1.0,1.0,1.0,1.0,0.2,0.647,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001041,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.991,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001042,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7947,1,0.0,1.0,1.0,1.0,1.0,0.7,0.785,1.0,TKV change from baseline at 36 months |
| HLT011-001043,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7795,1,0.4,1.0,1.0,1.0,0.0,1.0,0.911,1.0,TKV change from baseline at 36 months |
| HLT011-001044,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8035,1,0.4,1.0,1.0,0.0,1.0,1.0,0.966,1.0,TKV change from baseline at 36 months |
| HLT011-001045,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6413,1,1.0,1.0,0.3,0.0,1.0,0.2,0.836,1.0,Enzyme activity normalization at 12 months |
| HLT011-001046,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5801,0,0.0,0.0,1.0,1.0,1.0,1.0,0.822,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001047,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.823,1,1.0,1.0,1.0,0.0,1.0,0.2,0.748,1.0,6MWT distance change at 48 weeks |
| HLT011-001048,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9808,1,1.0,1.0,1.0,1.0,1.0,0.7,0.864,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001049,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.6133,1,1.0,0.0,1.0,1.0,0.0,0.7,0.827,1.0,6MWT distance change at 48 weeks |
| HLT011-001050,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9608,1,1.0,1.0,1.0,1.0,1.0,0.7,0.767,1.0,Motor milestone achievement at 14 months |
| HLT011-001051,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7955,1,1.0,1.0,1.0,0.0,1.0,0.2,0.964,1.0,TKV change from baseline at 36 months |
| HLT011-001052,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8206,1,1.0,1.0,1.0,0.0,1.0,1.0,0.901,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001053,Friedreich Ataxia,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5759,0,0.0,0.0,1.0,1.0,1.0,0.2,0.965,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001054,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6398,1,0.4,1.0,0.3,0.0,1.0,1.0,0.774,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001055,Rett Syndrome,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4221,0,0.0,0.0,1.0,0.0,1.0,1.0,0.561,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001056,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7517,1,0.4,1.0,1.0,0.0,1.0,1.0,0.909,1.0,TKV change from baseline at 36 months |
| HLT011-001057,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.6698,1,1.0,0.0,1.0,1.0,1.0,1.0,0.978,1.0,Enzyme activity normalization at 12 months |
| HLT011-001058,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7014,1,1.0,1.0,0.3,1.0,0.0,0.7,0.826,1.0,6MWT distance change at 48 weeks |
| HLT011-001059,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7933,1,0.0,1.0,1.0,1.0,1.0,1.0,0.922,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001060,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7661,1,1.0,1.0,1.0,0.0,1.0,0.7,0.852,1.0,TKV change from baseline at 36 months |
| HLT011-001061,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5629,0,1.0,0.0,1.0,0.0,1.0,1.0,0.958,1.0,MLMT performance score improvement at 1 year |
| HLT011-001062,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4441,0,0.0,0.0,0.3,1.0,1.0,0.7,0.914,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001063,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9264,1,1.0,1.0,1.0,1.0,0.0,0.7,0.988,1.0,Motor milestone achievement at 14 months |
| HLT011-001064,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6213,1,1.0,0.0,0.3,1.0,1.0,1.0,0.966,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001065,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5575,0,1.0,0.0,1.0,0.0,1.0,0.7,0.883,1.0,Motor milestone achievement at 14 months |
| HLT011-001066,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6009,1,1.0,0.0,1.0,0.0,1.0,0.7,0.906,1.0,Aortic root z-score change at 36 months |
| HLT011-001067,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5229,0,1.0,0.0,0.0,1.0,1.0,0.5,0.033,1.0,Aortic root z-score change at 36 months |
| HLT011-001068,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8083,1,1.0,1.0,0.3,1.0,1.0,0.2,0.921,1.0,TKV change from baseline at 36 months |
| HLT011-001069,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.907,1.0,Enzyme activity normalization at 12 months |
| HLT011-001070,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6636,1,0.4,0.0,1.0,1.0,1.0,0.7,0.952,1.0,Aortic root z-score change at 36 months |
| HLT011-001071,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.4131,0,0.0,0.0,1.0,0.0,1.0,0.7,0.72,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001072,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.2576,0,0.0,0.0,0.3,0.0,1.0,0.7,0.839,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001073,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7496,1,1.0,0.0,1.0,1.0,1.0,1.0,0.899,1.0,Aortic root z-score change at 36 months |
| HLT011-001074,Down Syndrome,Chromosomal,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6692,1,0.4,0.0,1.0,1.0,1.0,1.0,0.98,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001075,Li-Fraumeni Syndrome,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.3987,0,0.0,0.0,0.0,1.0,1.0,1.0,0.93,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001076,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2628,0,0.0,0.0,0.0,0.0,1.0,1.0,0.856,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001077,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7772,1,0.4,1.0,1.0,0.0,1.0,1.0,0.956,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001078,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7453,1,0.0,1.0,1.0,0.0,1.0,1.0,0.958,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001079,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6683,1,0.4,0.0,1.0,1.0,1.0,1.0,0.0,1.0,TKV change from baseline at 36 months |
| HLT011-001080,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6003,1,1.0,0.0,0.0,1.0,1.0,1.0,0.992,1.0,Motor milestone achievement at 14 months |
| HLT011-001081,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9769,1,1.0,1.0,1.0,1.0,1.0,1.0,0.762,1.0,Motor milestone achievement at 14 months |
| HLT011-001082,Down Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6213,1,0.0,0.0,1.0,1.0,1.0,1.0,0.666,1.0,TKV change from baseline at 36 months |
| HLT011-001083,Down Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5773,1,1.0,0.0,1.0,0.0,1.0,1.0,0.655,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001084,Usher Syndrome Type 1,Rare auditory,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.1555,0,0.4,0.0,0.0,0.0,0.0,1.0,0.991,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001085,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4194,0,1.0,0.0,0.3,0.0,1.0,0.2,0.886,1.0,Aortic root z-score change at 36 months |
| HLT011-001086,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.95,1,1.0,1.0,1.0,1.0,1.0,1.0,0.894,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001087,Epidermolysis Bullosa,Rare dermatological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4916,0,0.0,0.0,1.0,0.0,1.0,1.0,0.87,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001088,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.4717,0,0.4,0.0,1.0,0.0,1.0,0.7,0.919,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001089,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6971,1,1.0,0.0,1.0,1.0,1.0,0.2,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001090,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4364,0,1.0,0.0,0.3,0.0,1.0,1.0,0.94,1.0,Aortic root z-score change at 36 months |
| HLT011-001091,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.7466,1,1.0,1.0,0.0,1.0,1.0,0.2,0.914,1.0,Motor milestone achievement at 14 months |
| HLT011-001092,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7183,1,0.0,1.0,0.3,1.0,1.0,1.0,0.501,1.0,TKV change from baseline at 36 months |
| HLT011-001093,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9832,1,1.0,1.0,1.0,1.0,1.0,0.7,0.878,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001094,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.7493,1,0.0,1.0,1.0,0.0,1.0,1.0,0.903,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001095,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4205,0,0.0,0.0,1.0,0.0,1.0,0.7,0.9,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001096,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.489,0,1.0,0.0,0.3,0.0,1.0,1.0,0.966,1.0,Aortic root z-score change at 36 months |
| HLT011-001097,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8504,1,1.0,1.0,0.3,1.0,1.0,1.0,0.584,1.0,6MWT distance change at 48 weeks |
| HLT011-001098,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5387,0,1.0,0.0,0.0,1.0,1.0,0.2,0.894,1.0,MLMT performance score improvement at 1 year |
| HLT011-001099,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4581,0,1.0,0.0,0.3,0.0,1.0,1.0,0.988,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001100,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6559,1,0.4,1.0,1.0,0.0,0.0,1.0,0.977,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001101,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9731,1,1.0,1.0,1.0,1.0,1.0,1.0,0.939,1.0,Enzyme activity normalization at 12 months |
| HLT011-001102,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3113,0,0.0,0.0,0.3,0.0,1.0,0.7,0.783,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001103,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8638,1,1.0,1.0,0.3,1.0,1.0,0.7,0.876,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001104,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6171,1,1.0,0.0,1.0,0.0,1.0,1.0,0.976,1.0,MLMT performance score improvement at 1 year |
| HLT011-001105,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9043,1,1.0,1.0,0.3,1.0,1.0,1.0,0.941,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001106,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8606,1,1.0,1.0,1.0,0.0,1.0,0.7,0.306,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001107,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9853,1,1.0,1.0,1.0,1.0,1.0,1.0,0.885,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001108,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7479,1,1.0,0.0,1.0,1.0,1.0,0.7,0.958,1.0,Aortic root z-score change at 36 months |
| HLT011-001109,Propionic Acidemia,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7385,1,1.0,0.0,1.0,1.0,1.0,0.7,0.995,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001110,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7464,1,1.0,1.0,0.0,1.0,1.0,0.7,0.214,1.0,6MWT distance change at 48 weeks |
| HLT011-001111,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8179,1,0.4,1.0,0.3,1.0,1.0,1.0,0.976,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001112,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6589,1,1.0,0.0,1.0,1.0,0.0,1.0,0.933,1.0,Aortic root z-score change at 36 months |
| HLT011-001113,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.3522,0,0.0,0.0,0.3,1.0,0.0,1.0,0.959,1.0,TKV change from baseline at 36 months |
| HLT011-001114,Down Syndrome,Chromosomal,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5191,0,0.0,0.0,1.0,1.0,1.0,0.7,0.875,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001115,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5177,0,1.0,0.0,1.0,0.0,1.0,0.7,0.671,1.0,Aortic root z-score change at 36 months |
| HLT011-001116,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6717,1,0.4,0.0,1.0,1.0,1.0,0.7,0.996,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001117,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7336,1,0.4,1.0,1.0,0.0,1.0,0.7,0.46,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001118,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8478,1,1.0,1.0,0.3,1.0,1.0,1.0,0.342,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001119,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6597,1,0.0,1.0,1.0,0.0,1.0,1.0,0.984,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001120,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5197,0,1.0,0.0,0.3,0.0,1.0,1.0,0.986,1.0,MLMT performance score improvement at 1 year |
| HLT011-001121,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8447,1,1.0,1.0,1.0,0.0,1.0,1.0,0.0,1.0,TKV change from baseline at 36 months |
| HLT011-001122,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.968,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001123,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4446,0,1.0,0.0,0.3,0.0,1.0,1.0,0.926,1.0,MLMT performance score improvement at 1 year |
| HLT011-001124,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.724,1,1.0,1.0,1.0,0.0,0.0,0.7,0.929,1.0,6MWT distance change at 48 weeks |
| HLT011-001125,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5805,1,1.0,0.0,1.0,1.0,0.0,1.0,0.813,1.0,Aortic root z-score change at 36 months |
| HLT011-001126,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6547,1,0.0,1.0,0.0,1.0,1.0,1.0,0.899,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001127,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8571,1,0.0,1.0,1.0,1.0,1.0,0.7,0.792,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001128,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5958,1,1.0,1.0,0.0,0.0,1.0,1.0,0.771,1.0,TKV change from baseline at 36 months |
| HLT011-001129,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9791,1,1.0,1.0,1.0,1.0,1.0,1.0,0.966,1.0,TKV change from baseline at 36 months |
| HLT011-001130,Alagille Syndrome,Rare hepatic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4633,0,1.0,0.0,1.0,0.0,0.0,1.0,0.934,1.0,MLMT performance score improvement at 1 year |
| HLT011-001131,Propionic Acidemia,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7384,1,1.0,0.0,1.0,1.0,1.0,0.7,0.961,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001132,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5368,0,0.0,1.0,1.0,0.0,0.0,1.0,0.979,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001133,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8537,1,1.0,1.0,1.0,0.0,1.0,0.7,0.908,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001134,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9121,1,1.0,1.0,1.0,1.0,0.0,1.0,0.65,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001135,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.578,1,1.0,0.0,1.0,0.0,1.0,0.7,0.917,1.0,Aortic root z-score change at 36 months |
| HLT011-001136,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3851,0,0.0,0.0,0.3,1.0,1.0,1.0,0.984,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001137,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6774,1,1.0,0.0,1.0,1.0,1.0,0.2,0.644,1.0,Aortic root z-score change at 36 months |
| HLT011-001138,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7733,1,1.0,0.0,1.0,1.0,1.0,0.7,0.944,1.0,Aortic root z-score change at 36 months |
| HLT011-001139,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6328,1,1.0,0.0,1.0,1.0,0.0,1.0,0.775,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001140,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.704,1,0.0,1.0,1.0,1.0,0.0,1.0,0.648,1.0,TKV change from baseline at 36 months |
| HLT011-001141,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4222,0,0.0,0.0,0.0,1.0,1.0,1.0,0.936,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001142,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7744,1,1.0,1.0,0.0,1.0,1.0,1.0,0.822,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001143,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4391,0,1.0,0.0,0.3,0.0,1.0,0.2,0.965,1.0,MLMT performance score improvement at 1 year |
| HLT011-001144,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.743,1,1.0,0.0,1.0,1.0,1.0,0.5,0.869,1.0,MLMT performance score improvement at 1 year |
| HLT011-001145,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5527,1,1.0,0.0,1.0,0.0,0.0,1.0,0.921,1.0,Aortic root z-score change at 36 months |
| HLT011-001146,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5019,0,1.0,0.0,0.3,1.0,0.0,0.7,0.755,1.0,Motor milestone achievement at 14 months |
| HLT011-001147,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.7102,1,0.4,1.0,1.0,0.0,1.0,1.0,0.824,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001148,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6226,1,1.0,0.0,1.0,0.0,1.0,0.7,0.962,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001149,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5087,0,0.0,1.0,0.0,1.0,0.0,0.7,0.785,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001150,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.581,1,1.0,0.0,1.0,0.0,1.0,1.0,0.928,1.0,Enzyme activity normalization at 12 months |
| HLT011-001151,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8964,1,0.4,1.0,1.0,1.0,1.0,0.7,0.968,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001152,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5398,0,1.0,0.0,0.0,1.0,1.0,1.0,0.431,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001153,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8952,1,1.0,1.0,1.0,1.0,0.0,0.2,0.94,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001154,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9142,1,0.4,1.0,1.0,1.0,1.0,1.0,0.847,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001155,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7991,1,0.0,1.0,1.0,1.0,1.0,1.0,0.894,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001156,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.589,1,0.0,0.0,1.0,1.0,1.0,1.0,0.758,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001157,MPS I (Hurler),Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.3916,0,0.0,0.0,1.0,0.0,1.0,1.0,0.932,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001158,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.9441,1,1.0,1.0,1.0,1.0,1.0,1.0,0.21,1.0,Enzyme activity normalization at 12 months |
| HLT011-001159,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5615,1,1.0,0.0,0.0,1.0,1.0,0.7,0.907,1.0,Aortic root z-score change at 36 months |
| HLT011-001160,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.8557,1,1.0,1.0,1.0,0.0,1.0,1.0,0.919,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001161,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8486,1,0.0,1.0,1.0,1.0,1.0,1.0,0.541,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001162,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8333,1,0.4,1.0,1.0,1.0,0.0,0.2,0.982,1.0,TKV change from baseline at 36 months |
| HLT011-001163,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.742,1,1.0,0.0,1.0,1.0,1.0,1.0,0.266,1.0,MLMT performance score improvement at 1 year |
| HLT011-001164,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.793,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001165,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9293,1,0.4,1.0,1.0,1.0,1.0,0.2,0.0,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001166,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7288,1,1.0,0.0,1.0,1.0,1.0,1.0,0.507,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001167,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6123,1,1.0,0.0,1.0,1.0,0.0,0.7,0.94,1.0,MLMT performance score improvement at 1 year |
| HLT011-001168,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7113,1,0.0,1.0,1.0,0.0,1.0,1.0,0.949,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001169,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7371,1,1.0,0.0,1.0,1.0,1.0,1.0,0.131,1.0,MLMT performance score improvement at 1 year |
| HLT011-001170,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8304,1,1.0,1.0,1.0,0.0,1.0,1.0,0.873,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001171,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.617,1,1.0,0.0,1.0,1.0,0.0,1.0,0.931,1.0,Aortic root z-score change at 36 months |
| HLT011-001172,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.975,1,0.4,1.0,1.0,1.0,1.0,1.0,0.857,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001173,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.9924,1,1.0,1.0,1.0,1.0,1.0,0.7,0.821,1.0,Motor milestone achievement at 14 months |
| HLT011-001174,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.4973,0,0.4,0.0,0.3,1.0,1.0,1.0,0.977,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001175,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8539,1,0.0,1.0,1.0,1.0,1.0,1.0,0.215,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001176,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6444,1,0.4,0.0,1.0,1.0,1.0,0.7,0.989,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001177,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7398,1,1.0,0.0,1.0,1.0,1.0,1.0,0.914,1.0,Aortic root z-score change at 36 months |
| HLT011-001178,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.8454,1,1.0,1.0,0.3,1.0,1.0,0.7,0.35,1.0,Aortic root z-score change at 36 months |
| HLT011-001179,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8544,1,0.4,1.0,1.0,1.0,1.0,0.7,0.839,1.0,6MWT distance change at 48 weeks |
| HLT011-001180,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9188,1,0.4,1.0,1.0,1.0,1.0,1.0,0.939,1.0,6MWT distance change at 48 weeks |
| HLT011-001181,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.426,0,0.4,0.0,0.3,1.0,0.0,0.2,0.353,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001182,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9193,1,0.4,1.0,1.0,1.0,1.0,1.0,0.843,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001183,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5632,1,1.0,0.0,0.3,1.0,1.0,0.7,0.347,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001184,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8364,1,1.0,1.0,1.0,0.0,1.0,1.0,0.956,1.0,6MWT distance change at 48 weeks |
| HLT011-001185,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7875,1,1.0,1.0,0.0,1.0,1.0,1.0,0.837,1.0,6MWT distance change at 48 weeks |
| HLT011-001186,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7351,1,1.0,0.0,1.0,1.0,1.0,1.0,0.882,1.0,MLMT performance score improvement at 1 year |
| HLT011-001187,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5941,1,1.0,0.0,1.0,0.0,1.0,0.2,0.955,1.0,Aortic root z-score change at 36 months |
| HLT011-001188,Alagille Syndrome,Rare hepatic,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5307,0,0.0,0.0,1.0,1.0,1.0,0.7,0.823,1.0,TKV change from baseline at 36 months |
| HLT011-001189,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5223,0,0.4,1.0,0.3,0.0,0.0,1.0,0.215,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001190,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8875,1,1.0,1.0,1.0,1.0,0.0,1.0,0.964,1.0,TKV change from baseline at 36 months |
| HLT011-001191,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7398,1,1.0,0.0,1.0,1.0,1.0,0.5,0.95,1.0,Aortic root z-score change at 36 months |
| HLT011-001192,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5252,0,1.0,0.0,1.0,0.0,1.0,0.2,0.699,1.0,Aortic root z-score change at 36 months |
| HLT011-001193,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.617,1,1.0,0.0,1.0,0.0,1.0,0.5,0.989,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001194,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5651,1,0.4,0.0,1.0,1.0,0.0,0.7,0.611,1.0,Aortic root z-score change at 36 months |
| HLT011-001195,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.3827,0,0.0,0.0,1.0,0.0,1.0,0.5,0.939,1.0,Motor milestone achievement at 14 months |
| HLT011-001196,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8243,1,1.0,1.0,0.0,1.0,1.0,1.0,0.981,1.0,6MWT distance change at 48 weeks |
| HLT011-001197,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7449,1,0.4,1.0,0.3,1.0,1.0,0.2,0.824,1.0,6MWT distance change at 48 weeks |
| HLT011-001198,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8454,1,0.4,1.0,1.0,1.0,1.0,0.7,0.853,1.0,TKV change from baseline at 36 months |
| HLT011-001199,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.8557,1,1.0,1.0,0.3,1.0,1.0,1.0,0.881,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001200,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9779,1,1.0,1.0,1.0,1.0,1.0,0.2,0.965,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001201,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6441,1,0.0,1.0,0.0,1.0,1.0,0.7,0.648,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001202,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8618,1,1.0,1.0,0.3,1.0,1.0,1.0,0.883,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001203,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7554,1,1.0,0.0,1.0,1.0,1.0,1.0,0.888,1.0,MLMT performance score improvement at 1 year |
| HLT011-001204,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.944,1.0,TKV change from baseline at 36 months |
| HLT011-001205,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,0.5,0.892,1.0,Enzyme activity normalization at 12 months |
| HLT011-001206,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6141,1,0.4,0.0,1.0,1.0,1.0,0.2,0.854,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001207,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4651,0,0.0,0.0,1.0,1.0,0.0,1.0,0.0,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001208,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6944,1,1.0,0.0,1.0,1.0,1.0,1.0,0.9,1.0,Aortic root z-score change at 36 months |
| HLT011-001209,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.8062,1,0.0,1.0,1.0,1.0,1.0,0.2,0.511,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001210,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7804,1,1.0,0.0,1.0,1.0,1.0,1.0,0.763,1.0,Aortic root z-score change at 36 months |
| HLT011-001211,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.5649,1,1.0,0.0,1.0,0.0,1.0,1.0,0.915,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001212,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5964,0,1.0,0.0,1.0,0.0,1.0,0.7,0.943,1.0,MLMT performance score improvement at 1 year |
| HLT011-001213,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4615,0,0.0,1.0,0.0,0.0,1.0,1.0,0.634,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001214,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6969,1,1.0,0.0,1.0,1.0,1.0,0.2,0.97,1.0,Aortic root z-score change at 36 months |
| HLT011-001215,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.682,1,0.0,1.0,0.0,1.0,1.0,0.7,0.99,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001216,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7473,1,0.4,1.0,0.3,1.0,1.0,1.0,0.787,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001217,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5446,0,1.0,0.0,0.3,1.0,1.0,1.0,0.691,1.0,Motor milestone achievement at 14 months |
| HLT011-001218,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7646,1,0.4,1.0,0.0,1.0,1.0,1.0,0.956,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001219,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8932,1,0.4,1.0,1.0,1.0,1.0,0.2,0.903,1.0,TKV change from baseline at 36 months |
| HLT011-001220,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7319,1,1.0,0.0,1.0,1.0,1.0,0.7,0.981,1.0,Enzyme activity normalization at 12 months |
| HLT011-001221,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.974,1.0,Enzyme activity normalization at 12 months |
| HLT011-001222,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8377,1,0.4,1.0,1.0,1.0,1.0,1.0,0.0,1.0,6MWT distance change at 48 weeks |
| HLT011-001223,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5031,0,1.0,0.0,0.0,1.0,1.0,0.2,0.828,1.0,Enzyme activity normalization at 12 months |
| HLT011-001224,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.832,1,1.0,1.0,0.3,1.0,1.0,0.5,0.937,1.0,TKV change from baseline at 36 months |
| HLT011-001225,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7402,1,0.4,1.0,0.3,1.0,1.0,0.7,0.898,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001226,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.5811,1,0.0,0.0,1.0,1.0,1.0,1.0,0.951,1.0,6MWT distance change at 48 weeks |
| HLT011-001227,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5232,0,0.4,0.0,1.0,0.0,1.0,1.0,0.744,1.0,Aortic root z-score change at 36 months |
| HLT011-001228,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.2145,0,0.4,0.0,0.0,0.0,0.0,1.0,0.894,1.0,Aortic root z-score change at 36 months |
| HLT011-001229,Down Syndrome,Chromosomal,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.4864,0,0.4,0.0,0.3,1.0,1.0,0.2,0.955,1.0,6MWT distance change at 48 weeks |
| HLT011-001230,Niemann-Pick Type C,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.9027,1,0.4,1.0,1.0,1.0,1.0,0.7,0.643,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001231,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9537,1,1.0,1.0,1.0,1.0,1.0,0.2,0.941,1.0,6MWT distance change at 48 weeks |
| HLT011-001232,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7015,1,0.0,1.0,0.0,1.0,1.0,1.0,0.487,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001233,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9118,1,1.0,1.0,1.0,1.0,1.0,0.2,0.695,1.0,6MWT distance change at 48 weeks |
| HLT011-001234,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8747,1,1.0,1.0,1.0,0.0,1.0,0.7,0.963,1.0,TKV change from baseline at 36 months |
| HLT011-001235,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.979,1,1.0,1.0,1.0,1.0,1.0,0.2,0.0,1.0,6MWT distance change at 48 weeks |
| HLT011-001236,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7011,1,0.4,1.0,1.0,0.0,1.0,0.2,0.957,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001237,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9568,1,1.0,1.0,1.0,1.0,0.0,1.0,0.665,1.0,6MWT distance change at 48 weeks |
| HLT011-001238,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5163,0,1.0,0.0,1.0,0.0,0.0,0.7,0.947,1.0,Aortic root z-score change at 36 months |
| HLT011-001239,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8525,1,1.0,1.0,0.3,1.0,1.0,0.2,0.846,1.0,6MWT distance change at 48 weeks |
| HLT011-001240,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9781,1,1.0,1.0,1.0,1.0,1.0,0.5,0.907,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001241,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3436,0,1.0,0.0,0.3,0.0,0.0,1.0,0.662,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001242,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7185,1,1.0,0.0,1.0,1.0,1.0,0.2,0.625,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001243,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4211,0,0.0,0.0,1.0,0.0,1.0,0.2,0.923,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001244,MPS I (Hurler),Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.568,1,1.0,0.0,1.0,0.0,1.0,1.0,0.845,1.0,Enzyme activity normalization at 12 months |
| HLT011-001245,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.763,1,1.0,0.0,1.0,1.0,1.0,0.7,0.912,1.0,Motor milestone achievement at 14 months |
| HLT011-001246,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6956,1,0.0,1.0,0.3,1.0,1.0,0.2,0.932,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001247,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5893,0,0.0,0.0,1.0,1.0,1.0,0.2,0.963,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001248,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7499,1,1.0,1.0,0.0,1.0,1.0,0.7,0.0,1.0,6MWT distance change at 48 weeks |
| HLT011-001249,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7237,1,1.0,0.0,1.0,1.0,1.0,1.0,0.991,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001250,Down Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.4645,0,0.4,0.0,0.3,1.0,1.0,1.0,0.785,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001251,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9145,1,0.4,1.0,1.0,1.0,1.0,1.0,0.913,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001252,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9966,1,1.0,1.0,1.0,1.0,1.0,1.0,0.971,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001253,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8932,1,0.4,1.0,1.0,1.0,1.0,1.0,0.88,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001254,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.2072,0,0.0,0.0,0.0,0.0,1.0,1.0,0.82,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001255,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8029,1,0.4,1.0,0.3,1.0,1.0,1.0,0.975,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001256,Li-Fraumeni Syndrome,Rare oncological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.5771,1,1.0,0.0,1.0,0.0,1.0,1.0,0.902,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001257,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.6674,1,0.4,0.0,1.0,1.0,1.0,1.0,0.886,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001258,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7014,1,1.0,0.0,1.0,1.0,1.0,1.0,0.77,1.0,MLMT performance score improvement at 1 year |
| HLT011-001259,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7183,1,0.4,1.0,0.0,1.0,1.0,0.2,0.905,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001260,Glycogen Storage Disease I,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5655,1,0.4,0.0,1.0,1.0,0.0,1.0,0.893,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001261,Pompe Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5277,0,0.0,0.0,0.3,1.0,1.0,1.0,0.931,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001262,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7902,1,1.0,0.0,1.0,1.0,1.0,1.0,0.825,1.0,Aortic root z-score change at 36 months |
| HLT011-001263,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5817,0,0.0,0.0,1.0,1.0,1.0,1.0,0.984,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001264,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7384,1,1.0,0.0,1.0,1.0,1.0,1.0,0.939,1.0,MLMT performance score improvement at 1 year |
| HLT011-001265,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6457,1,0.0,1.0,1.0,0.0,1.0,0.2,0.363,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001266,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7254,1,1.0,1.0,1.0,0.0,0.0,0.7,0.974,1.0,MLMT performance score improvement at 1 year |
| HLT011-001267,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9367,1,1.0,1.0,1.0,1.0,0.0,1.0,0.758,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001268,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7089,1,1.0,0.0,1.0,1.0,1.0,0.2,0.812,1.0,Aortic root z-score change at 36 months |
| HLT011-001269,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8771,1,0.0,1.0,1.0,1.0,1.0,0.2,0.672,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001270,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4476,0,0.0,1.0,0.3,0.0,0.0,0.7,0.897,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001271,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7736,1,1.0,0.0,1.0,1.0,1.0,1.0,0.0,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001272,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7457,1,1.0,0.0,1.0,1.0,1.0,0.7,0.899,1.0,Aortic root z-score change at 36 months |
| HLT011-001273,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4948,0,1.0,0.0,1.0,0.0,0.0,0.2,0.931,1.0,Aortic root z-score change at 36 months |
| HLT011-001274,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8092,1,0.0,1.0,1.0,1.0,1.0,0.7,0.893,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001275,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7734,1,0.4,1.0,1.0,0.0,1.0,1.0,0.988,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001276,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7294,1,1.0,0.0,1.0,1.0,1.0,0.2,0.392,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001277,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.976,1.0,6MWT distance change at 48 weeks |
| HLT011-001278,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8204,1,1.0,1.0,0.3,1.0,1.0,1.0,0.965,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001279,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6888,1,1.0,0.0,1.0,1.0,1.0,0.7,0.787,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001280,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4455,0,0.0,0.0,1.0,1.0,0.0,1.0,0.952,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001281,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.4253,0,0.0,0.0,0.0,1.0,1.0,1.0,0.719,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001282,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3988,0,1.0,0.0,0.0,0.0,1.0,1.0,0.974,1.0,Aortic root z-score change at 36 months |
| HLT011-001283,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5811,1,1.0,0.0,1.0,1.0,0.0,1.0,0.964,1.0,Aortic root z-score change at 36 months |
| HLT011-001284,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6417,1,0.4,0.0,1.0,1.0,1.0,0.7,0.98,1.0,TKV change from baseline at 36 months |
| HLT011-001285,Down Syndrome,Chromosomal,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5153,0,1.0,0.0,0.0,1.0,1.0,1.0,0.007,1.0,Motor milestone achievement at 14 months |
| HLT011-001286,Down Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4143,0,0.0,0.0,1.0,1.0,0.0,0.7,0.0,1.0,TKV change from baseline at 36 months |
| HLT011-001287,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7242,1,1.0,0.0,1.0,1.0,1.0,0.7,0.932,1.0,Aortic root z-score change at 36 months |
| HLT011-001288,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6149,1,1.0,0.0,0.3,1.0,1.0,0.2,0.524,1.0,Motor milestone achievement at 14 months |
| HLT011-001289,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4682,0,1.0,0.0,0.3,0.0,1.0,1.0,0.825,1.0,Aortic root z-score change at 36 months |
| HLT011-001290,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7264,1,0.4,1.0,0.0,1.0,1.0,1.0,0.985,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001291,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4501,0,1.0,0.0,0.3,0.0,1.0,0.7,0.956,1.0,Aortic root z-score change at 36 months |
| HLT011-001292,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7019,1,0.0,1.0,1.0,0.0,1.0,1.0,0.733,1.0,TKV change from baseline at 36 months |
| HLT011-001293,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8007,1,0.4,1.0,1.0,1.0,0.0,0.2,0.978,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001294,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.775,1,1.0,0.0,1.0,1.0,1.0,1.0,0.956,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001295,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6706,1,1.0,0.0,1.0,1.0,1.0,0.2,0.794,1.0,Motor milestone achievement at 14 months |
| HLT011-001296,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6054,1,1.0,0.0,0.3,1.0,1.0,0.7,0.919,1.0,Aortic root z-score change at 36 months |
| HLT011-001297,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6288,1,0.4,1.0,1.0,0.0,0.0,0.2,0.849,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001298,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8664,1,1.0,1.0,1.0,1.0,0.0,0.7,0.897,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001299,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9789,1,1.0,1.0,1.0,1.0,1.0,0.5,0.986,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001300,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6893,1,1.0,0.0,1.0,1.0,1.0,0.7,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001301,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9584,1,1.0,1.0,1.0,1.0,1.0,1.0,0.686,1.0,6MWT distance change at 48 weeks |
| HLT011-001302,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8384,1,0.0,1.0,1.0,1.0,1.0,1.0,0.967,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001303,Achondroplasia,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.4434,0,0.4,0.0,1.0,0.0,1.0,0.2,0.95,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001304,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.887,1.0,Motor milestone achievement at 14 months |
| HLT011-001305,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7006,1,1.0,0.0,1.0,1.0,1.0,1.0,0.503,1.0,MLMT performance score improvement at 1 year |
| HLT011-001306,Down Syndrome,Chromosomal,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.6825,1,1.0,0.0,1.0,1.0,1.0,0.5,0.599,1.0,Motor milestone achievement at 14 months |
| HLT011-001307,Beta-Thalassemia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8201,1,0.0,1.0,1.0,1.0,1.0,0.7,0.968,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001308,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6398,1,0.4,1.0,1.0,0.0,0.0,1.0,0.953,1.0,TKV change from baseline at 36 months |
| HLT011-001309,MPS I (Hurler),Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5772,1,0.0,0.0,1.0,1.0,1.0,0.2,0.975,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001310,Down Syndrome,Chromosomal,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5897,1,0.0,0.0,1.0,1.0,1.0,1.0,0.868,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001311,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5231,0,1.0,0.0,0.0,1.0,1.0,0.2,0.67,1.0,Aortic root z-score change at 36 months |
| HLT011-001312,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7471,1,1.0,0.0,1.0,1.0,1.0,1.0,0.947,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001313,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4588,0,0.0,0.0,0.0,1.0,1.0,1.0,0.962,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001314,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.949,1.0,Enzyme activity normalization at 12 months |
| HLT011-001315,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7997,1,0.0,1.0,1.0,1.0,1.0,0.2,0.321,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001316,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9124,1,0.4,1.0,1.0,1.0,1.0,0.7,0.931,1.0,TKV change from baseline at 36 months |
| HLT011-001317,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7831,1,0.4,1.0,0.3,1.0,1.0,0.7,0.94,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001318,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8262,1,1.0,1.0,1.0,1.0,0.0,0.7,0.938,1.0,Enzyme activity normalization at 12 months |
| HLT011-001319,Osteogenesis Imperfecta,Rare skeletal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.9244,1,1.0,1.0,1.0,1.0,1.0,0.5,0.831,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001320,Li-Fraumeni Syndrome,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5961,1,1.0,0.0,1.0,1.0,0.0,0.2,0.984,1.0,TKV change from baseline at 36 months |
| HLT011-001321,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.5631,0,0.0,1.0,0.3,0.0,1.0,0.2,0.858,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001322,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5866,1,1.0,0.0,0.3,1.0,1.0,0.7,0.934,1.0,Aortic root z-score change at 36 months |
| HLT011-001323,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.8007,1,1.0,1.0,0.3,1.0,1.0,1.0,0.958,1.0,Enzyme activity normalization at 12 months |
| HLT011-001324,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9608,1,1.0,1.0,1.0,1.0,1.0,1.0,0.96,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001325,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6628,1,1.0,0.0,1.0,1.0,1.0,1.0,0.845,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001326,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.626,1,1.0,0.0,1.0,1.0,0.0,0.7,0.788,1.0,Aortic root z-score change at 36 months |
| HLT011-001327,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.9862,1,1.0,1.0,1.0,1.0,1.0,1.0,0.965,1.0,Aortic root z-score change at 36 months |
| HLT011-001328,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.939,1,0.4,1.0,1.0,1.0,1.0,1.0,0.889,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001329,Huntington Disease,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6969,1,0.0,1.0,1.0,0.0,1.0,0.2,0.764,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001330,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9771,1,1.0,1.0,1.0,1.0,1.0,0.7,0.806,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001331,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7712,1,1.0,1.0,1.0,0.0,1.0,0.7,0.909,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001332,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.8407,1,0.0,1.0,1.0,1.0,1.0,0.7,0.473,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001333,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5631,1,0.0,1.0,0.3,0.0,1.0,0.7,0.949,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001334,Angelman Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7589,1,1.0,0.0,1.0,1.0,1.0,1.0,0.987,1.0,MLMT performance score improvement at 1 year |
| HLT011-001335,Prader-Willi Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5422,0,0.0,0.0,1.0,1.0,1.0,0.5,0.906,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001336,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6876,1,0.4,0.0,1.0,1.0,1.0,0.2,0.978,1.0,TKV change from baseline at 36 months |
| HLT011-001337,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.9325,1,1.0,1.0,1.0,1.0,1.0,0.2,0.776,1.0,6MWT distance change at 48 weeks |
| HLT011-001338,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.3723,0,0.0,0.0,1.0,0.0,1.0,1.0,0.096,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001339,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5421,0,0.0,0.0,1.0,1.0,1.0,1.0,0.0,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001340,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6852,1,0.4,1.0,1.0,0.0,0.0,0.7,0.725,1.0,Aortic root z-score change at 36 months |
| HLT011-001341,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7495,1,0.4,1.0,1.0,0.0,1.0,1.0,0.912,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001342,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.8735,1,1.0,1.0,1.0,0.0,1.0,1.0,0.945,1.0,MLMT performance score improvement at 1 year |
| HLT011-001343,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5907,1,1.0,0.0,1.0,0.0,1.0,0.7,0.854,1.0,Aortic root z-score change at 36 months |
| HLT011-001344,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6145,1,0.0,1.0,0.0,1.0,1.0,0.2,0.987,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001345,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6898,1,1.0,0.0,1.0,1.0,0.0,1.0,0.962,1.0,Aortic root z-score change at 36 months |
| HLT011-001346,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5886,1,0.4,0.0,1.0,1.0,1.0,0.7,0.8,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001347,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5719,1,1.0,0.0,1.0,0.0,1.0,1.0,0.865,1.0,Aortic root z-score change at 36 months |
| HLT011-001348,Marfan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.85,1.0,Aortic root z-score change at 36 months |
| HLT011-001349,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5029,0,1.0,0.0,0.3,1.0,0.0,1.0,0.367,1.0,Aortic root z-score change at 36 months |
| HLT011-001350,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7904,1,1.0,1.0,1.0,0.0,1.0,0.7,0.98,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001351,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9753,1,1.0,1.0,1.0,1.0,1.0,1.0,0.9,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001352,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5687,0,1.0,0.0,0.3,1.0,1.0,0.7,0.604,1.0,MLMT performance score improvement at 1 year |
| HLT011-001353,Rett Syndrome,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.6917,1,1.0,0.0,1.0,1.0,1.0,0.2,0.49,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001354,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9447,1,1.0,1.0,1.0,1.0,1.0,0.7,0.933,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001355,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7137,1,1.0,0.0,1.0,1.0,1.0,0.7,0.92,1.0,Enzyme activity normalization at 12 months |
| HLT011-001356,Gaucher Disease Type 1,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.788,1.0,Enzyme activity normalization at 12 months |
| HLT011-001357,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8711,1,1.0,1.0,0.3,1.0,1.0,0.7,0.962,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001358,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.3811,0,1.0,0.0,0.0,0.0,1.0,0.7,0.915,1.0,Aortic root z-score change at 36 months |
| HLT011-001359,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.6663,1,0.0,1.0,1.0,0.0,1.0,0.5,0.934,1.0,TKV change from baseline at 36 months |
| HLT011-001360,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9902,1,1.0,1.0,1.0,1.0,1.0,0.7,0.933,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001361,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8069,1,0.0,1.0,1.0,1.0,1.0,0.7,0.779,1.0,TKV change from baseline at 36 months |
| HLT011-001362,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6934,1,1.0,0.0,1.0,1.0,1.0,0.5,0.928,1.0,Aortic root z-score change at 36 months |
| HLT011-001363,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.4581,0,1.0,0.0,1.0,0.0,0.0,0.7,0.945,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001364,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8261,1,0.0,1.0,1.0,1.0,1.0,1.0,0.839,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001365,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4551,0,0.4,0.0,0.3,1.0,1.0,1.0,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001366,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8419,1,0.0,1.0,1.0,1.0,1.0,0.7,0.969,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001367,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,1.0,0.836,1.0,6MWT distance change at 48 weeks |
| HLT011-001368,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8588,1,0.4,1.0,1.0,1.0,1.0,1.0,0.88,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001369,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8762,1,0.4,1.0,1.0,1.0,1.0,0.2,0.916,1.0,6MWT distance change at 48 weeks |
| HLT011-001370,Neurofibromatosis Type 1,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7441,1,1.0,0.0,1.0,1.0,1.0,0.7,0.861,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001371,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7602,1,0.4,1.0,0.3,1.0,1.0,1.0,0.685,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001372,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4636,0,0.4,0.0,1.0,0.0,1.0,1.0,0.908,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001373,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6116,1,1.0,0.0,1.0,1.0,0.0,1.0,0.984,1.0,Aortic root z-score change at 36 months |
| HLT011-001374,Leber Congenital Amaurosis,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6959,1,1.0,1.0,0.3,0.0,1.0,0.7,0.902,1.0,MLMT performance score improvement at 1 year |
| HLT011-001375,Kabuki Syndrome,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.5302,0,0.0,0.0,1.0,1.0,1.0,1.0,0.887,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001376,Phenylketonuria,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.5976,1,0.0,0.0,1.0,1.0,1.0,0.7,0.974,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001377,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.87,1,1.0,1.0,1.0,0.0,1.0,0.7,0.876,1.0,TKV change from baseline at 36 months |
| HLT011-001378,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.6054,1,1.0,0.0,0.3,1.0,1.0,0.5,0.963,1.0,MLMT performance score improvement at 1 year |
| HLT011-001379,Alport Syndrome,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4061,0,0.0,0.0,1.0,0.0,1.0,0.5,0.905,1.0,TKV change from baseline at 36 months |
| HLT011-001380,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5919,1,1.0,0.0,1.0,0.0,1.0,1.0,0.849,1.0,Aortic root z-score change at 36 months |
| HLT011-001381,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5988,1,1.0,0.0,1.0,1.0,0.0,1.0,0.913,1.0,Aortic root z-score change at 36 months |
| HLT011-001382,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.7662,1,0.0,1.0,1.0,1.0,1.0,0.7,0.953,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001383,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.7512,1,0.4,1.0,1.0,0.0,1.0,0.5,0.429,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001384,Alagille Syndrome,Rare hepatic,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4121,0,0.0,0.0,0.3,1.0,1.0,0.7,0.789,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001385,Turner Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.2954,0,0.0,0.0,1.0,0.0,0.0,0.7,0.967,1.0,TKV change from baseline at 36 months |
| HLT011-001386,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8044,1,0.0,1.0,1.0,1.0,1.0,1.0,0.889,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001387,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8962,1,0.4,1.0,1.0,1.0,1.0,1.0,0.712,1.0,TKV change from baseline at 36 months |
| HLT011-001388,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.9657,1,1.0,1.0,1.0,1.0,1.0,0.7,0.986,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001389,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4582,0,1.0,0.0,0.3,0.0,1.0,0.7,0.963,1.0,Aortic root z-score change at 36 months |
| HLT011-001390,Pompe Disease,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7561,1,1.0,0.0,1.0,1.0,1.0,1.0,0.882,1.0,Enzyme activity normalization at 12 months |
| HLT011-001391,Down Syndrome,Chromosomal,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6669,1,0.4,0.0,1.0,1.0,1.0,1.0,0.99,1.0,Aortic root z-score change at 36 months |
| HLT011-001392,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7946,1,1.0,0.0,1.0,1.0,1.0,1.0,0.909,1.0,MLMT performance score improvement at 1 year |
| HLT011-001393,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7075,1,0.4,1.0,1.0,0.0,1.0,0.5,0.983,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001394,Niemann-Pick Type C,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.9905,1,1.0,1.0,1.0,1.0,1.0,1.0,0.737,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001395,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4659,0,1.0,0.0,0.0,1.0,0.0,0.7,0.798,1.0,MLMT performance score improvement at 1 year |
| HLT011-001396,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6889,1,0.4,1.0,1.0,0.0,0.0,1.0,0.7,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001397,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6965,1,0.4,1.0,1.0,0.0,1.0,0.5,0.97,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001398,Down Syndrome,Chromosomal,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.5325,0,0.4,0.0,1.0,0.0,1.0,0.7,0.793,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001399,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.862,1,0.0,1.0,1.0,1.0,1.0,1.0,0.893,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001400,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5878,1,1.0,0.0,1.0,0.0,1.0,0.7,0.797,1.0,Aortic root z-score change at 36 months |
| HLT011-001401,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4521,0,1.0,0.0,0.3,0.0,1.0,1.0,0.956,1.0,Aortic root z-score change at 36 months |
| HLT011-001402,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.5649,1,1.0,0.0,0.3,1.0,1.0,0.2,0.949,1.0,Enzyme activity normalization at 12 months |
| HLT011-001403,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-OI-001,Phase 2/3,Setrusumab anti-sclerostin vs bisphosphonate,0.4105,0,0.4,0.0,0.3,1.0,0.0,1.0,0.937,1.0,Lumbar spine BMD change at 24 months |
| HLT011-001404,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5465,0,1.0,0.0,0.0,1.0,1.0,1.0,0.926,1.0,Motor milestone achievement at 14 months |
| HLT011-001405,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7803,1,1.0,1.0,0.0,1.0,1.0,0.7,0.664,1.0,6MWT distance change at 48 weeks |
| HLT011-001406,Tuberous Sclerosis,Rare neurological,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.7359,1,0.4,1.0,1.0,0.0,1.0,1.0,0.875,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001407,Angelman Syndrome,Chromosomal,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4043,0,0.0,0.0,1.0,0.0,0.0,0.2,0.963,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001408,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.6565,1,1.0,1.0,0.3,0.0,1.0,1.0,0.994,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001409,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5131,0,0.4,0.0,0.3,1.0,1.0,0.7,0.848,1.0,Aortic root z-score change at 36 months |
| HLT011-001410,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.824,1,1.0,1.0,1.0,0.0,1.0,1.0,0.895,1.0,TKV change from baseline at 36 months |
| HLT011-001411,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7298,1,1.0,0.0,1.0,1.0,1.0,0.5,0.815,0.6,MLMT performance score improvement at 1 year |
| HLT011-001412,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5882,1,1.0,0.0,1.0,0.0,1.0,0.7,0.962,1.0,Aortic root z-score change at 36 months |
| HLT011-001413,Usher Syndrome Type 1,Rare auditory,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6335,1,1.0,0.0,1.0,0.0,1.0,1.0,0.941,1.0,Aortic root z-score change at 36 months |
| HLT011-001414,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.4836,0,0.0,0.0,1.0,1.0,0.0,0.5,0.929,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001415,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7934,1,0.4,1.0,1.0,0.0,1.0,1.0,0.98,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001416,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7841,1,1.0,1.0,0.3,1.0,1.0,1.0,0.814,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001417,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8644,1,0.0,1.0,1.0,1.0,1.0,0.2,0.956,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001418,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6596,1,0.0,1.0,0.0,1.0,1.0,1.0,0.946,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001419,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.686,1,1.0,0.0,1.0,1.0,1.0,0.5,0.755,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001420,22q11.2 Deletion Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.4343,0,1.0,0.0,0.3,0.0,1.0,1.0,0.973,1.0,MLMT performance score improvement at 1 year |
| HLT011-001421,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8516,1,0.0,1.0,1.0,1.0,1.0,1.0,0.908,1.0,TKV change from baseline at 36 months |
| HLT011-001422,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5933,1,1.0,0.0,0.3,1.0,1.0,0.7,0.861,1.0,Aortic root z-score change at 36 months |
| HLT011-001423,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7576,1,1.0,0.0,1.0,1.0,1.0,1.0,0.971,1.0,Aortic root z-score change at 36 months |
| HLT011-001424,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5962,1,1.0,0.0,1.0,0.0,1.0,0.2,0.855,1.0,Aortic root z-score change at 36 months |
| HLT011-001425,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5626,0,1.0,0.0,0.0,1.0,1.0,1.0,0.446,1.0,Motor milestone achievement at 14 months |
| HLT011-001426,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5072,0,0.4,0.0,1.0,0.0,1.0,1.0,0.897,1.0,Aortic root z-score change at 36 months |
| HLT011-001427,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.7522,1,0.4,1.0,1.0,0.0,1.0,1.0,0.943,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001428,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5894,1,1.0,0.0,1.0,1.0,0.0,0.7,0.85,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001429,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9799,1,1.0,1.0,1.0,1.0,1.0,0.2,0.894,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001430,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.6721,1,1.0,0.0,1.0,1.0,1.0,1.0,0.817,1.0,Aortic root z-score change at 36 months |
| HLT011-001431,Down Syndrome,Chromosomal,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7045,1,1.0,0.0,1.0,1.0,1.0,1.0,0.977,1.0,Aortic root z-score change at 36 months |
| HLT011-001432,Hereditary Hemorrhagic Telangectasia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.4446,0,0.0,0.0,1.0,0.0,1.0,1.0,0.767,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001433,Fabry Disease,Rare metabolic,TRIAL-FAB-001,Phase 3,Agalsidase beta ERT vs Migalastat chaperone,0.8438,1,0.0,1.0,1.0,1.0,1.0,1.0,0.913,1.0,GFR stabilization and lyso-Gb3 reduction at 24 months |
| HLT011-001434,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8648,1,1.0,1.0,1.0,0.0,1.0,1.0,0.322,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001435,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8436,1,1.0,1.0,1.0,0.0,1.0,1.0,0.965,1.0,6MWT distance change at 48 weeks |
| HLT011-001436,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5172,0,1.0,0.0,1.0,0.0,1.0,0.2,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001437,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.5148,0,0.4,1.0,0.0,0.0,1.0,1.0,0.0,1.0,6MWT distance change at 48 weeks |
| HLT011-001438,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.744,1,0.0,1.0,0.3,1.0,1.0,1.0,0.861,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001439,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5796,1,0.0,0.0,1.0,1.0,1.0,1.0,0.706,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001440,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5649,1,1.0,0.0,1.0,0.0,1.0,0.7,0.467,1.0,Aortic root z-score change at 36 months |
| HLT011-001441,Spinal Muscular Atrophy,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.8092,1,1.0,1.0,0.3,1.0,1.0,1.0,0.873,1.0,Motor milestone achievement at 14 months |
| HLT011-001442,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7344,1,1.0,0.0,1.0,1.0,1.0,0.7,0.491,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001443,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.8318,1,0.4,1.0,1.0,1.0,1.0,0.5,0.818,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001444,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7104,1,1.0,0.0,1.0,1.0,1.0,0.2,0.618,1.0,Enzyme activity normalization at 12 months |
| HLT011-001445,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8326,1,1.0,1.0,0.0,1.0,1.0,0.2,0.765,1.0,6MWT distance change at 48 weeks |
| HLT011-001446,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5236,0,0.4,1.0,0.0,0.0,1.0,1.0,0.886,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001447,Down Syndrome,Chromosomal,TRIAL-TS-001,Phase 3,Everolimus mTOR inhibitor,0.4821,0,1.0,0.0,0.3,1.0,0.0,0.7,0.572,1.0,SEGA volume reduction ≥50% at 9 months |
| HLT011-001448,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.5894,1,0.0,1.0,0.3,0.0,1.0,0.5,0.85,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001449,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5275,0,1.0,0.0,0.0,1.0,1.0,1.0,0.948,1.0,Motor milestone achievement at 14 months |
| HLT011-001450,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8044,1,1.0,1.0,0.0,1.0,1.0,1.0,0.923,1.0,6MWT distance change at 48 weeks |
| HLT011-001451,Multiple Endocrine Neoplasia 2,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.7438,1,1.0,0.0,1.0,1.0,1.0,1.0,0.793,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001452,Primary Immunodeficiency (CVID),Rare immunological,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.6679,1,0.4,0.0,1.0,1.0,1.0,1.0,0.981,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001453,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7337,1,1.0,0.0,1.0,1.0,1.0,0.7,0.947,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001454,Down Syndrome,Chromosomal,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.3798,0,0.0,0.0,0.3,1.0,1.0,0.2,0.992,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001455,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6099,1,0.0,0.0,1.0,1.0,1.0,1.0,0.881,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001456,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9405,1,1.0,1.0,1.0,1.0,1.0,0.2,0.309,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001457,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7496,1,1.0,1.0,0.0,1.0,1.0,1.0,0.934,1.0,6MWT distance change at 48 weeks |
| HLT011-001458,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.619,1,1.0,0.0,0.3,1.0,1.0,0.5,0.978,1.0,Aortic root z-score change at 36 months |
| HLT011-001459,Down Syndrome,Chromosomal,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.5966,0,1.0,0.0,1.0,0.0,1.0,0.7,0.923,1.0,MLMT performance score improvement at 1 year |
| HLT011-001460,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5071,0,1.0,0.0,0.0,1.0,1.0,0.7,0.696,1.0,Aortic root z-score change at 36 months |
| HLT011-001461,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9731,1,1.0,1.0,1.0,1.0,1.0,0.7,0.785,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001462,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.6037,1,0.0,1.0,0.0,1.0,1.0,1.0,0.957,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001463,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8652,1,0.4,1.0,1.0,1.0,1.0,0.7,0.932,1.0,6MWT distance change at 48 weeks |
| HLT011-001464,Neurofibromatosis Type 1,Rare neurological,TRIAL-SMA-001,Phase 3,Nusinersen ASO vs Onasemnogene gene therapy,0.5787,0,1.0,0.0,0.0,1.0,1.0,1.0,0.728,1.0,Motor milestone achievement at 14 months |
| HLT011-001465,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7252,1,0.4,1.0,1.0,0.0,1.0,1.0,0.479,1.0,TKV change from baseline at 36 months |
| HLT011-001466,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8205,1,1.0,1.0,1.0,0.0,1.0,0.2,0.442,1.0,TKV change from baseline at 36 months |
| HLT011-001467,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.568,1,1.0,0.0,1.0,0.0,1.0,0.7,0.872,1.0,Aortic root z-score change at 36 months |
| HLT011-001468,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.8065,1,1.0,1.0,0.0,1.0,1.0,1.0,0.969,1.0,6MWT distance change at 48 weeks |
| HLT011-001469,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.4924,0,0.0,1.0,0.0,0.0,1.0,1.0,0.889,1.0,TKV change from baseline at 36 months |
| HLT011-001470,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,1.0,1,1.0,1.0,1.0,1.0,1.0,0.7,0.846,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001471,Down Syndrome,Chromosomal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.5832,1,0.0,0.0,1.0,1.0,1.0,1.0,0.975,1.0,TKV change from baseline at 36 months |
| HLT011-001472,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.7998,1,1.0,1.0,0.3,1.0,1.0,0.7,0.967,1.0,6MWT distance change at 48 weeks |
| HLT011-001473,Cystic Fibrosis,Rare pulmonary,TRIAL-CF-001,Phase 3,Elexacaftor/Tezacaftor/Ivacaftor triple therapy,0.9181,1,1.0,1.0,1.0,1.0,1.0,0.2,0.719,1.0,ppFEV1 change from baseline at 24 weeks |
| HLT011-001474,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.6707,1,0.0,1.0,1.0,0.0,1.0,0.7,0.955,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001475,Sickle Cell Disease,Rare hematological,TRIAL-SCD-001,Phase 3,Betibeglogene gene therapy vs Voxelotor,0.8115,1,0.0,1.0,1.0,1.0,1.0,1.0,0.965,1.0,VOC rate reduction ≥50% at 24 months |
| HLT011-001476,Hemophilia A,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7601,1,0.0,1.0,1.0,1.0,0.0,1.0,0.928,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001477,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5707,1,1.0,0.0,1.0,0.0,1.0,0.7,0.89,1.0,Aortic root z-score change at 36 months |
| HLT011-001478,Glycogen Storage Disease I,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5532,1,1.0,0.0,0.3,1.0,1.0,1.0,0.846,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001479,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7185,1,1.0,0.0,1.0,1.0,1.0,0.2,0.812,1.0,Aortic root z-score change at 36 months |
| HLT011-001480,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4133,0,1.0,0.0,0.3,0.0,1.0,0.7,0.972,1.0,Aortic root z-score change at 36 months |
| HLT011-001481,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4332,0,0.4,0.0,1.0,0.0,1.0,0.2,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001482,Phenylketonuria,Rare metabolic,TRIAL-GD-001,Phase 3,Imiglucerase ERT vs Eliglustat SRT,0.7383,1,1.0,0.0,1.0,1.0,1.0,0.7,0.867,1.0,Enzyme activity normalization at 12 months |
| HLT011-001483,Beta-Thalassemia,Rare hematological,TRIAL-HEM-001,Phase 3,Gene therapy (AAV5-F8) vs Factor VIII prophylaxis,0.7472,1,1.0,0.0,1.0,1.0,1.0,1.0,0.669,1.0,ABR reduction ≥80% from baseline at 12 months |
| HLT011-001484,Duchenne Muscular Dystrophy,Rare muscular,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.91,1,1.0,1.0,0.3,1.0,1.0,1.0,0.764,1.0,6MWT distance change at 48 weeks |
| HLT011-001485,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.89,1,0.4,1.0,1.0,1.0,1.0,1.0,0.788,1.0,TKV change from baseline at 36 months |
| HLT011-001486,Pompe Disease,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.5174,1,0.4,0.0,1.0,0.0,1.0,1.0,0.985,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001487,Noonan Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.7636,1,1.0,0.0,1.0,1.0,1.0,1.0,0.427,1.0,Aortic root z-score change at 36 months |
| HLT011-001488,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.4581,0,1.0,0.0,1.0,0.0,0.0,0.2,0.835,1.0,Aortic root z-score change at 36 months |
| HLT011-001489,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.6814,1,1.0,0.0,1.0,1.0,1.0,1.0,0.99,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001490,Long QT Syndrome,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.5772,1,1.0,0.0,1.0,0.0,1.0,0.7,0.0,1.0,Aortic root z-score change at 36 months |
| HLT011-001491,Phenylketonuria,Rare metabolic,TRIAL-NPC-001,Phase 2/3,Arimoclomol HSP inducer,0.7408,1,1.0,0.0,1.0,1.0,1.0,1.0,0.808,1.0,NPC-CSS score stabilization at 12 months |
| HLT011-001492,Stargardt Disease,Rare ophthalmic,TRIAL-LCA-001,Phase 3,Voretigene neparvovec gene therapy,0.7175,1,1.0,0.0,1.0,1.0,1.0,1.0,0.928,1.0,MLMT performance score improvement at 1 year |
| HLT011-001493,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.8494,1,1.0,1.0,0.3,1.0,1.0,1.0,0.519,1.0,TKV change from baseline at 36 months |
| HLT011-001494,Congenital Adrenal Hyperplasia,Rare endocrine,TRIAL-CAH-001,Phase 3,Crinecerfont (CRF1 receptor antagonist),0.9757,1,1.0,1.0,1.0,1.0,1.0,1.0,0.773,1.0,Androstenedione normalization at 24 weeks |
| HLT011-001495,Hypertrophic Cardiomyopathy,Rare cardiac,TRIAL-MFS-001,Phase 3,Losartan vs Atenolol for aortic root dilation,0.732,1,1.0,0.0,1.0,1.0,1.0,1.0,0.929,1.0,Aortic root z-score change at 36 months |
| HLT011-001496,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.9037,1,1.0,1.0,1.0,1.0,0.0,0.7,0.918,1.0,TKV change from baseline at 36 months |
| HLT011-001497,Hereditary Breast/Ovarian Ca,Rare oncological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.6581,1,1.0,0.0,1.0,1.0,0.0,0.7,0.622,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001498,Wiskott-Aldrich Syndrome,Rare immunological,TRIAL-DMD-001,Phase 2/3,Exon 51 skipping ASO therapy,0.4945,0,1.0,0.0,0.0,1.0,1.0,0.7,0.948,1.0,6MWT distance change at 48 weeks |
| HLT011-001499,Neurofibromatosis Type 1,Rare neurological,TRIAL-HD-001,Phase 2/3,HTT-lowering ASO intrathecal therapy,0.3719,0,0.0,0.0,1.0,0.0,0.0,0.2,0.874,1.0,mHTT concentration reduction in CSF at 13 months |
| HLT011-001500,ADPKD,Rare renal,TRIAL-PKD-001,Phase 3,Tolvaptan vs placebo for TKV reduction,0.7529,1,0.4,1.0,1.0,0.0,1.0,0.5,0.302,1.0,TKV change from baseline at 36 months |
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