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HLT011-000150,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,0,24,24,Enzyme activity normalization at 12 months,1
HLT011-001244,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,4,24,28,Enzyme activity normalization at 12 months,1
HLT011-000423,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,10,24,34,Enzyme activity normalization at 12 months,1
HLT011-000052,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,11,24,35,Enzyme activity normalization at 12 months,1
HLT011-000643,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,8,24,32,Enzyme activity normalization at 12 months,1
HLT011-000998,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,2,24,26,Enzyme activity normalization at 12 months,1
HLT011-000265,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,5,24,29,Enzyme activity normalization at 12 months,1
HLT011-000743,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,9,24,33,Enzyme activity normalization at 12 months,1
HLT011-000281,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,3,24,27,Enzyme activity normalization at 12 months,1
HLT011-001057,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,9,24,33,Enzyme activity normalization at 12 months,1
HLT011-000872,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,10,24,34,Enzyme activity normalization at 12 months,1
HLT011-000668,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,7,24,31,Enzyme activity normalization at 12 months,1
HLT011-000239,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,10,24,34,Enzyme activity normalization at 12 months,1
HLT011-000841,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,2,24,26,Enzyme activity normalization at 12 months,1
HLT011-000176,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,3,24,27,Enzyme activity normalization at 12 months,1
HLT011-000568,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,7,24,31,Enzyme activity normalization at 12 months,1
HLT011-001101,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,10,24,34,Enzyme activity normalization at 12 months,1
HLT011-000988,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,2,24,26,Enzyme activity normalization at 12 months,1
HLT011-000741,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,3,24,27,Enzyme activity normalization at 12 months,1
HLT011-000775,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,5,24,29,Enzyme activity normalization at 12 months,1
HLT011-001205,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,7,24,31,Enzyme activity normalization at 12 months,1
HLT011-000395,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,3,24,27,Enzyme activity normalization at 12 months,1
HLT011-000744,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,8,24,32,Enzyme activity normalization at 12 months,1
HLT011-000826,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Eliglustat,7,24,31,Enzyme activity normalization at 12 months,1
HLT011-001402,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,0,24,24,Enzyme activity normalization at 12 months,1
HLT011-000205,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,5,24,29,Enzyme activity normalization at 12 months,1
HLT011-001069,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,1,24,25,Enzyme activity normalization at 12 months,1
HLT011-000811,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,9,24,33,Enzyme activity normalization at 12 months,1
HLT011-000647,TRIAL-GD-001,Gaucher Disease Type 1,Rare metabolic,Imiglucerase,8,24,32,Enzyme activity normalization at 12 months,1
HLT011-000074,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,6,24,30,Motor milestone achievement at 14 months,1
HLT011-000169,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,5,24,29,Motor milestone achievement at 14 months,1
HLT011-000781,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,11,24,35,Motor milestone achievement at 14 months,1
HLT011-000267,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,5,24,29,Motor milestone achievement at 14 months,1
HLT011-000971,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,11,24,35,Motor milestone achievement at 14 months,1
HLT011-001245,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,8,24,32,Motor milestone achievement at 14 months,1
HLT011-001306,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,8,24,32,Motor milestone achievement at 14 months,1
HLT011-000656,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,5,24,29,Motor milestone achievement at 14 months,1
HLT011-001050,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,6,24,30,Motor milestone achievement at 14 months,1
HLT011-000255,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,5,24,29,Motor milestone achievement at 14 months,1
HLT011-001080,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,7,24,31,Motor milestone achievement at 14 months,1
HLT011-000160,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,2,24,26,Motor milestone achievement at 14 months,1
HLT011-000830,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,7,24,31,Motor milestone achievement at 14 months,1
HLT011-000747,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,0,24,24,Motor milestone achievement at 14 months,1
HLT011-000200,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,6,24,30,Motor milestone achievement at 14 months,1
HLT011-000348,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,8,24,32,Motor milestone achievement at 14 months,1
HLT011-000173,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,11,24,35,Motor milestone achievement at 14 months,1
HLT011-000163,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,1,24,25,Motor milestone achievement at 14 months,1
HLT011-001288,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,4,24,28,Motor milestone achievement at 14 months,1
HLT011-000427,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,7,24,31,Motor milestone achievement at 14 months,1
HLT011-000727,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,3,24,27,Motor milestone achievement at 14 months,1
HLT011-001081,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,4,24,28,Motor milestone achievement at 14 months,1
HLT011-000953,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,2,24,26,Motor milestone achievement at 14 months,1
HLT011-001295,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,10,24,34,Motor milestone achievement at 14 months,1
HLT011-000284,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,5,24,29,Motor milestone achievement at 14 months,1
HLT011-000206,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Onasemnogene,10,24,34,Motor milestone achievement at 14 months,1
HLT011-000693,TRIAL-SMA-001,Spinal Muscular Atrophy,Rare neurological,Nusinersen,8,24,32,Motor milestone achievement at 14 months,1
HLT011-001193,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,3,24,27,ppFEV1 change from baseline at 24 weeks,1
HLT011-001003,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-001165,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000413,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,4,24,28,ppFEV1 change from baseline at 24 weeks,1
HLT011-000849,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,11,24,35,ppFEV1 change from baseline at 24 weeks,1
HLT011-000230,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,8,24,32,ppFEV1 change from baseline at 24 weeks,1
HLT011-001415,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-000389,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000894,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,11,24,35,ppFEV1 change from baseline at 24 weeks,1
HLT011-001278,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000161,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-000762,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,7,24,31,ppFEV1 change from baseline at 24 weeks,1
HLT011-000128,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000516,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000204,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,7,24,31,ppFEV1 change from baseline at 24 weeks,1
HLT011-000231,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-001166,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,4,24,28,ppFEV1 change from baseline at 24 weeks,1
HLT011-000925,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-001368,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000129,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-000619,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000155,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000099,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-000086,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,3,24,27,ppFEV1 change from baseline at 24 weeks,1
HLT011-000240,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,7,24,31,ppFEV1 change from baseline at 24 weeks,1
HLT011-001396,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-000883,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-000417,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-001182,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,10,24,34,ppFEV1 change from baseline at 24 weeks,1
HLT011-001443,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-001251,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-000679,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-000462,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-000750,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000029,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000845,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,8,24,32,ppFEV1 change from baseline at 24 weeks,1
HLT011-000350,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-000335,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-000538,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,8,24,32,ppFEV1 change from baseline at 24 weeks,1
HLT011-001371,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,10,24,34,ppFEV1 change from baseline at 24 weeks,1
HLT011-000209,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-001253,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-001041,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,4,24,28,ppFEV1 change from baseline at 24 weeks,1
HLT011-000815,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-001170,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,4,24,28,ppFEV1 change from baseline at 24 weeks,1
HLT011-000268,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,3,24,27,ppFEV1 change from baseline at 24 weeks,1
HLT011-000733,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-000193,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,7,24,31,ppFEV1 change from baseline at 24 weeks,1
HLT011-001133,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-000523,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,0,24,24,ppFEV1 change from baseline at 24 weeks,1
HLT011-000869,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-000091,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,8,24,32,ppFEV1 change from baseline at 24 weeks,1
HLT011-000510,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,2,24,26,ppFEV1 change from baseline at 24 weeks,1
HLT011-000764,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,6,24,30,ppFEV1 change from baseline at 24 weeks,1
HLT011-000060,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,5,24,29,ppFEV1 change from baseline at 24 weeks,1
HLT011-000217,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,ETI-triple,1,24,25,ppFEV1 change from baseline at 24 weeks,1
HLT011-000636,TRIAL-CF-001,Cystic Fibrosis,Rare pulmonary,Placebo,9,24,33,ppFEV1 change from baseline at 24 weeks,1
HLT011-000716,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,6,18,24,6MWT distance change at 48 weeks,1
HLT011-000016,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,11,18,29,6MWT distance change at 48 weeks,1
HLT011-000482,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,8,18,26,6MWT distance change at 48 weeks,1
HLT011-000737,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,11,18,29,6MWT distance change at 48 weeks,1
HLT011-000436,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,2,18,20,6MWT distance change at 48 weeks,1
HLT011-000663,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,6,18,24,6MWT distance change at 48 weeks,1
HLT011-000196,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,0,18,18,6MWT distance change at 48 weeks,1
HLT011-001124,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,7,18,25,6MWT distance change at 48 weeks,1
HLT011-000051,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,7,18,25,6MWT distance change at 48 weeks,1
HLT011-000213,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,6,18,24,6MWT distance change at 48 weeks,1
HLT011-001049,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,8,18,26,6MWT distance change at 48 weeks,1
HLT011-000767,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,0,18,18,6MWT distance change at 48 weeks,1
HLT011-001277,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,6,18,24,6MWT distance change at 48 weeks,1
HLT011-000851,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,5,18,23,6MWT distance change at 48 weeks,1
HLT011-001301,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,11,18,29,6MWT distance change at 48 weeks,1
HLT011-000980,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,11,18,29,6MWT distance change at 48 weeks,1
HLT011-001445,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,11,18,29,6MWT distance change at 48 weeks,1
HLT011-001231,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,2,18,20,6MWT distance change at 48 weeks,1
HLT011-001184,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,7,18,25,6MWT distance change at 48 weeks,1
HLT011-000898,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,11,18,29,6MWT distance change at 48 weeks,1
HLT011-000143,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,11,18,29,6MWT distance change at 48 weeks,1
HLT011-001463,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,10,18,28,6MWT distance change at 48 weeks,1
HLT011-000071,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,9,18,27,6MWT distance change at 48 weeks,1
HLT011-001016,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,2,18,20,6MWT distance change at 48 weeks,1
HLT011-000791,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,1,18,19,6MWT distance change at 48 weeks,1
HLT011-001239,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,3,18,21,6MWT distance change at 48 weeks,1
HLT011-000936,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,6,18,24,6MWT distance change at 48 weeks,1
HLT011-001180,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,1,18,19,6MWT distance change at 48 weeks,1
HLT011-000419,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,Placebo,10,18,28,6MWT distance change at 48 weeks,1
HLT011-000885,TRIAL-DMD-001,Duchenne Muscular Dystrophy,Rare muscular,ASO-active,4,18,22,6MWT distance change at 48 weeks,1
HLT011-000190,TRIAL-HD-001,Huntington Disease,Rare neurological,HTT-ASO,0,24,24,mHTT concentration reduction in CSF at 13 months,1
HLT011-000309,TRIAL-HD-001,Huntington Disease,Rare neurological,HTT-ASO,10,24,34,mHTT concentration reduction in CSF at 13 months,1
HLT011-000941,TRIAL-HD-001,Huntington Disease,Rare neurological,Placebo,0,24,24,mHTT concentration reduction in CSF at 13 months,1
HLT011-001455,TRIAL-HD-001,Huntington Disease,Rare neurological,Placebo,5,24,29,mHTT concentration reduction in CSF at 13 months,1
HLT011-001329,TRIAL-HD-001,Huntington Disease,Rare neurological,HTT-ASO,2,24,26,mHTT concentration reduction in CSF at 13 months,1
HLT011-001215,TRIAL-HD-001,Huntington Disease,Rare neurological,Placebo,1,24,25,mHTT concentration reduction in CSF at 13 months,1
HLT011-001364,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Migalastat,1,24,25,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-001386,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,0,24,24,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000271,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,2,24,26,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000633,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,5,24,29,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-001309,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Migalastat,3,24,27,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000972,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,3,24,27,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000415,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Migalastat,3,24,27,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-001019,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Migalastat,4,24,28,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000912,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,4,24,28,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000664,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,0,24,24,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000723,TRIAL-FAB-001,Fabry Disease,Rare metabolic,Agalsidase-beta,3,24,27,GFR stabilization and lyso-Gb3 reduction at 24 months,1
HLT011-000141,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000384,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,7,36,43,Aortic root z-score change at 36 months,1
HLT011-001214,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,6,36,42,Aortic root z-score change at 36 months,1
HLT011-000253,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-000601,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,1,36,37,Aortic root z-score change at 36 months,1
HLT011-000119,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001066,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,0,36,36,Aortic root z-score change at 36 months,1
HLT011-001479,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000996,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001262,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,3,36,39,Aortic root z-score change at 36 months,1
HLT011-000187,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-001400,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000366,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,5,36,41,Aortic root z-score change at 36 months,1
HLT011-001039,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,9,36,45,Aortic root z-score change at 36 months,1
HLT011-000111,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,4,36,40,Aortic root z-score change at 36 months,1
HLT011-001006,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-001013,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,5,36,41,Aortic root z-score change at 36 months,1
HLT011-000754,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-000070,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,5,36,41,Aortic root z-score change at 36 months,1
HLT011-001028,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-000138,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,1,36,37,Aortic root z-score change at 36 months,1
HLT011-000599,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000547,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001431,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,6,36,42,Aortic root z-score change at 36 months,1
HLT011-001458,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,5,36,41,Aortic root z-score change at 36 months,1
HLT011-001340,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-001038,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000592,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,8,36,44,Aortic root z-score change at 36 months,1
HLT011-000847,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000487,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000250,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,10,36,46,Aortic root z-score change at 36 months,1
HLT011-001137,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,10,36,46,Aortic root z-score change at 36 months,1
HLT011-001287,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000276,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,2,36,38,Aortic root z-score change at 36 months,1
HLT011-000088,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-000373,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-001373,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-001194,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000914,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001380,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,3,36,39,Aortic root z-score change at 36 months,1
HLT011-000315,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-000981,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,1,36,37,Aortic root z-score change at 36 months,1
HLT011-000785,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000614,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-001272,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000011,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,6,36,42,Aortic root z-score change at 36 months,1
HLT011-000932,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,5,36,41,Aortic root z-score change at 36 months,1
HLT011-001089,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-001347,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,8,36,44,Aortic root z-score change at 36 months,1
HLT011-000447,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000680,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000497,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000232,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,8,36,44,Aortic root z-score change at 36 months,1
HLT011-001345,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,6,36,42,Aortic root z-score change at 36 months,1
HLT011-001138,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,1,36,37,Aortic root z-score change at 36 months,1
HLT011-000945,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,9,36,45,Aortic root z-score change at 36 months,1
HLT011-000709,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,2,36,38,Aortic root z-score change at 36 months,1
HLT011-000311,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-000874,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000166,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,11,36,47,Aortic root z-score change at 36 months,1
HLT011-001296,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,1,36,37,Aortic root z-score change at 36 months,1
HLT011-000901,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,9,36,45,Aortic root z-score change at 36 months,1
HLT011-000623,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,0,36,36,Aortic root z-score change at 36 months,1
HLT011-001424,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,10,36,46,Aortic root z-score change at 36 months,1
HLT011-000082,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000035,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000248,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001283,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Losartan,2,36,38,Aortic root z-score change at 36 months,1
HLT011-000027,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,2,36,38,Aortic root z-score change at 36 months,1
HLT011-000286,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,4,36,40,Aortic root z-score change at 36 months,1
HLT011-000877,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-001440,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,11,36,47,Aortic root z-score change at 36 months,1
HLT011-000676,TRIAL-MFS-001,Marfan Syndrome,Rare cardiac,Atenolol,3,36,39,Aortic root z-score change at 36 months,1
HLT011-001175,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,1,36,37,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000622,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,9,36,45,ABR reduction ≥80% from baseline at 12 months,1
HLT011-001078,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,0,36,36,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000907,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,11,36,47,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000652,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,5,36,41,ABR reduction ≥80% from baseline at 12 months,1
HLT011-001332,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,9,36,45,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000809,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,1,36,37,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000576,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,0,36,36,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000504,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,9,36,45,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000678,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,7,36,43,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000509,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,9,36,45,ABR reduction ≥80% from baseline at 12 months,1
HLT011-001476,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,3,36,39,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000126,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,7,36,43,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000304,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,5,36,41,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000730,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,10,36,46,ABR reduction ≥80% from baseline at 12 months,1
HLT011-001269,TRIAL-HEM-001,Hemophilia A,Rare hematological,Factor-VIII-prophylaxis,5,36,41,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000438,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,10,36,46,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000560,TRIAL-HEM-001,Hemophilia A,Rare hematological,AAV5-F8,10,36,46,ABR reduction ≥80% from baseline at 12 months,1
HLT011-000848,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,4,36,40,TKV change from baseline at 36 months,1
HLT011-000208,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,6,36,42,TKV change from baseline at 36 months,1
HLT011-001387,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,7,36,43,TKV change from baseline at 36 months,1
HLT011-000774,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,6,36,42,TKV change from baseline at 36 months,1
HLT011-001082,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,11,36,47,TKV change from baseline at 36 months,1
HLT011-000947,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,3,36,39,TKV change from baseline at 36 months,1
HLT011-000333,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,5,36,41,TKV change from baseline at 36 months,1
HLT011-000637,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,11,36,47,TKV change from baseline at 36 months,1
HLT011-001410,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,11,36,47,TKV change from baseline at 36 months,1
HLT011-000492,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,6,36,42,TKV change from baseline at 36 months,1
HLT011-000043,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,3,36,39,TKV change from baseline at 36 months,1
HLT011-001219,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,7,36,43,TKV change from baseline at 36 months,1
HLT011-000412,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,11,36,47,TKV change from baseline at 36 months,1
HLT011-001128,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,1,36,37,TKV change from baseline at 36 months,1
HLT011-000955,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,3,36,39,TKV change from baseline at 36 months,1
HLT011-000186,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,0,36,36,TKV change from baseline at 36 months,1
HLT011-001060,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,0,36,36,TKV change from baseline at 36 months,1
HLT011-000081,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,0,36,36,TKV change from baseline at 36 months,1
HLT011-000649,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,0,36,36,TKV change from baseline at 36 months,1
HLT011-000092,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,7,36,43,TKV change from baseline at 36 months,1
HLT011-000493,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,3,36,39,TKV change from baseline at 36 months,1
HLT011-000275,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,10,36,46,TKV change from baseline at 36 months,1
HLT011-000906,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,1,36,37,TKV change from baseline at 36 months,1
HLT011-000004,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,7,36,43,TKV change from baseline at 36 months,1
HLT011-000653,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,5,36,41,TKV change from baseline at 36 months,1
HLT011-000453,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,1,36,37,TKV change from baseline at 36 months,1
HLT011-000800,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,9,36,45,TKV change from baseline at 36 months,1
HLT011-000102,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,5,36,41,TKV change from baseline at 36 months,1
HLT011-001316,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,9,36,45,TKV change from baseline at 36 months,1
HLT011-001496,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,11,36,47,TKV change from baseline at 36 months,1
HLT011-000300,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,7,36,43,TKV change from baseline at 36 months,1
HLT011-000233,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,11,36,47,TKV change from baseline at 36 months,1
HLT011-000532,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,3,36,39,TKV change from baseline at 36 months,1
HLT011-001308,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,4,36,40,TKV change from baseline at 36 months,1
HLT011-001493,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,3,36,39,TKV change from baseline at 36 months,1
HLT011-000122,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,2,36,38,TKV change from baseline at 36 months,1
HLT011-001121,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,5,36,41,TKV change from baseline at 36 months,1
HLT011-000223,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,8,36,44,TKV change from baseline at 36 months,1
HLT011-000467,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,1,36,37,TKV change from baseline at 36 months,1
HLT011-000712,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,7,36,43,TKV change from baseline at 36 months,1
HLT011-001377,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,5,36,41,TKV change from baseline at 36 months,1
HLT011-000329,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,1,36,37,TKV change from baseline at 36 months,1
HLT011-000755,TRIAL-PKD-001,ADPKD,Rare renal,Placebo,4,36,40,TKV change from baseline at 36 months,1
HLT011-001092,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,10,36,46,TKV change from baseline at 36 months,1
HLT011-000078,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,11,36,47,TKV change from baseline at 36 months,1
HLT011-000368,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,4,36,40,TKV change from baseline at 36 months,1
HLT011-000736,TRIAL-PKD-001,ADPKD,Rare renal,Tolvaptan,10,36,46,TKV change from baseline at 36 months,1
HLT011-000007,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,0,24,24,Lumbar spine BMD change at 24 months,1
HLT011-000117,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,3,24,27,Lumbar spine BMD change at 24 months,1
HLT011-000837,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,7,24,31,Lumbar spine BMD change at 24 months,1
HLT011-000983,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,8,24,32,Lumbar spine BMD change at 24 months,1
HLT011-000331,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,6,24,30,Lumbar spine BMD change at 24 months,1
HLT011-001176,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,3,24,27,Lumbar spine BMD change at 24 months,1
HLT011-000799,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,7,24,31,Lumbar spine BMD change at 24 months,1
HLT011-001022,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,1,24,25,Lumbar spine BMD change at 24 months,1
HLT011-000256,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,10,24,34,Lumbar spine BMD change at 24 months,1
HLT011-001257,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,8,24,32,Lumbar spine BMD change at 24 months,1
HLT011-000992,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,5,24,29,Lumbar spine BMD change at 24 months,1
HLT011-001086,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,9,24,33,Lumbar spine BMD change at 24 months,1
HLT011-000697,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,5,24,29,Lumbar spine BMD change at 24 months,1
HLT011-000087,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,0,24,24,Lumbar spine BMD change at 24 months,1
HLT011-001147,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,1,24,25,Lumbar spine BMD change at 24 months,1
HLT011-000380,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,0,24,24,Lumbar spine BMD change at 24 months,1
HLT011-000180,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,8,24,32,Lumbar spine BMD change at 24 months,1
HLT011-000137,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Setrusumab,11,24,35,Lumbar spine BMD change at 24 months,1
HLT011-000871,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,7,24,31,Lumbar spine BMD change at 24 months,1
HLT011-000110,TRIAL-OI-001,Osteogenesis Imperfecta,Rare skeletal,Bisphosphonate,6,24,30,Lumbar spine BMD change at 24 months,1
HLT011-001093,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,10,18,28,SEGA volume reduction ≥50% at 9 months,1
HLT011-001325,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,5,18,23,SEGA volume reduction ≥50% at 9 months,1
HLT011-000044,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,5,18,23,SEGA volume reduction ≥50% at 9 months,1
HLT011-001353,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,8,18,26,SEGA volume reduction ≥50% at 9 months,1
HLT011-000157,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,8,18,26,SEGA volume reduction ≥50% at 9 months,1
HLT011-000965,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,5,18,23,SEGA volume reduction ≥50% at 9 months,1
HLT011-000401,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,10,18,28,SEGA volume reduction ≥50% at 9 months,1
HLT011-000330,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,3,18,21,SEGA volume reduction ≥50% at 9 months,1
HLT011-000588,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,3,18,21,SEGA volume reduction ≥50% at 9 months,1
HLT011-001370,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,7,18,25,SEGA volume reduction ≥50% at 9 months,1
HLT011-000669,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,7,18,25,SEGA volume reduction ≥50% at 9 months,1
HLT011-000277,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,8,18,26,SEGA volume reduction ≥50% at 9 months,1
HLT011-000956,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,0,18,18,SEGA volume reduction ≥50% at 9 months,1
HLT011-000351,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,10,18,28,SEGA volume reduction ≥50% at 9 months,1
HLT011-000700,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,0,18,18,SEGA volume reduction ≥50% at 9 months,1
HLT011-000705,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Placebo,3,18,21,SEGA volume reduction ≥50% at 9 months,1
HLT011-000077,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,9,18,27,SEGA volume reduction ≥50% at 9 months,1
HLT011-000464,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,10,18,28,SEGA volume reduction ≥50% at 9 months,1
HLT011-001294,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,5,18,23,SEGA volume reduction ≥50% at 9 months,1
HLT011-000225,TRIAL-TS-001,Tuberous Sclerosis,Rare neurological,Everolimus,6,18,24,SEGA volume reduction ≥50% at 9 months,1
HLT011-000640,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-000924,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,0,24,24,VOC rate reduction ≥50% at 24 months,1
HLT011-000079,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,2,24,26,VOC rate reduction ≥50% at 24 months,1
HLT011-000714,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000672,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,2,24,26,VOC rate reduction ≥50% at 24 months,1
HLT011-000961,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,7,24,31,VOC rate reduction ≥50% at 24 months,1
HLT011-000461,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-000228,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,0,24,24,VOC rate reduction ≥50% at 24 months,1
HLT011-000212,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-000690,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-000999,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-000827,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,1,24,25,VOC rate reduction ≥50% at 24 months,1
HLT011-000960,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,6,24,30,VOC rate reduction ≥50% at 24 months,1
HLT011-001328,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,1,24,25,VOC rate reduction ≥50% at 24 months,1
HLT011-000017,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-000328,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,8,24,32,VOC rate reduction ≥50% at 24 months,1
HLT011-000818,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-001126,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-001383,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-001274,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000559,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,2,24,26,VOC rate reduction ≥50% at 24 months,1
HLT011-000244,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-000496,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000951,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-001265,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,10,24,34,VOC rate reduction ≥50% at 24 months,1
HLT011-000882,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000360,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,0,24,24,VOC rate reduction ≥50% at 24 months,1
HLT011-001216,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,8,24,32,VOC rate reduction ≥50% at 24 months,1
HLT011-001155,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,2,24,26,VOC rate reduction ≥50% at 24 months,1
HLT011-001034,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000289,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-000490,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,10,24,34,VOC rate reduction ≥50% at 24 months,1
HLT011-000696,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-000183,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,11,24,35,VOC rate reduction ≥50% at 24 months,1
HLT011-000524,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,0,24,24,VOC rate reduction ≥50% at 24 months,1
HLT011-000829,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,1,24,25,VOC rate reduction ≥50% at 24 months,1
HLT011-000920,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,7,24,31,VOC rate reduction ≥50% at 24 months,1
HLT011-000483,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-001077,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-000130,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-001418,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-000969,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-000611,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000542,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-000303,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,10,24,34,VOC rate reduction ≥50% at 24 months,1
HLT011-001399,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-000537,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,8,24,32,VOC rate reduction ≥50% at 24 months,1
HLT011-001462,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,5,24,29,VOC rate reduction ≥50% at 24 months,1
HLT011-000148,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,1,24,25,VOC rate reduction ≥50% at 24 months,1
HLT011-000675,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000958,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-001344,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-000465,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,8,24,32,VOC rate reduction ≥50% at 24 months,1
HLT011-000336,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-001259,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,9,24,33,VOC rate reduction ≥50% at 24 months,1
HLT011-001302,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,3,24,27,VOC rate reduction ≥50% at 24 months,1
HLT011-000431,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Voxelotor,7,24,31,VOC rate reduction ≥50% at 24 months,1
HLT011-000165,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,4,24,28,VOC rate reduction ≥50% at 24 months,1
HLT011-001201,TRIAL-SCD-001,Sickle Cell Disease,Rare hematological,Betibeglogene,2,24,26,VOC rate reduction ≥50% at 24 months,1
HLT011-000370,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,5,18,23,NPC-CSS score stabilization at 12 months,1
HLT011-001478,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,10,18,28,NPC-CSS score stabilization at 12 months,1
HLT011-001489,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,6,18,24,NPC-CSS score stabilization at 12 months,1
HLT011-001442,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,6,18,24,NPC-CSS score stabilization at 12 months,1
HLT011-000511,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,6,18,24,NPC-CSS score stabilization at 12 months,1
HLT011-000949,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,11,18,29,NPC-CSS score stabilization at 12 months,1
HLT011-000911,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,5,18,23,NPC-CSS score stabilization at 12 months,1
HLT011-000903,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,5,18,23,NPC-CSS score stabilization at 12 months,1
HLT011-001083,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,4,18,22,NPC-CSS score stabilization at 12 months,1
HLT011-000548,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,1,18,19,NPC-CSS score stabilization at 12 months,1
HLT011-000361,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,1,18,19,NPC-CSS score stabilization at 12 months,1
HLT011-000132,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,0,18,18,NPC-CSS score stabilization at 12 months,1
HLT011-001131,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,1,18,19,NPC-CSS score stabilization at 12 months,1
HLT011-000280,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,2,18,20,NPC-CSS score stabilization at 12 months,1
HLT011-000075,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,1,18,19,NPC-CSS score stabilization at 12 months,1
HLT011-000235,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,10,18,28,NPC-CSS score stabilization at 12 months,1
HLT011-001271,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,0,18,18,NPC-CSS score stabilization at 12 months,1
HLT011-001394,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,8,18,26,NPC-CSS score stabilization at 12 months,1
HLT011-001486,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,0,18,18,NPC-CSS score stabilization at 12 months,1
HLT011-000449,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Placebo,9,18,27,NPC-CSS score stabilization at 12 months,1
HLT011-000293,TRIAL-NPC-001,Niemann-Pick Type C,Rare metabolic,Arimoclomol,5,18,23,NPC-CSS score stabilization at 12 months,1
HLT011-001334,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,3,12,15,MLMT performance score improvement at 1 year,1
HLT011-000892,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,8,12,20,MLMT performance score improvement at 1 year,1
HLT011-000982,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Sham-control,9,12,21,MLMT performance score improvement at 1 year,1
HLT011-001305,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,0,12,12,MLMT performance score improvement at 1 year,1
HLT011-001264,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,2,12,14,MLMT performance score improvement at 1 year,1
HLT011-001378,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,10,12,22,MLMT performance score improvement at 1 year,1
HLT011-000046,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,6,12,18,MLMT performance score improvement at 1 year,1
HLT011-000327,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,10,12,22,MLMT performance score improvement at 1 year,1
HLT011-000214,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,6,12,18,MLMT performance score improvement at 1 year,1
HLT011-000045,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,7,12,19,MLMT performance score improvement at 1 year,1
HLT011-000151,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,8,12,20,MLMT performance score improvement at 1 year,1
HLT011-001144,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Sham-control,9,12,21,MLMT performance score improvement at 1 year,1
HLT011-001186,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,7,12,19,MLMT performance score improvement at 1 year,1
HLT011-000667,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,9,12,21,MLMT performance score improvement at 1 year,1
HLT011-000543,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,1,12,13,MLMT performance score improvement at 1 year,1
HLT011-000334,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,11,12,23,MLMT performance score improvement at 1 year,1
HLT011-001392,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Sham-control,10,12,22,MLMT performance score improvement at 1 year,1
HLT011-000508,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,4,12,16,MLMT performance score improvement at 1 year,1
HLT011-000219,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,7,12,19,MLMT performance score improvement at 1 year,1
HLT011-000520,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Sham-control,10,12,22,MLMT performance score improvement at 1 year,1
HLT011-001169,TRIAL-LCA-001,Leber Congenital Amaurosis,Rare ophthalmic,Voretigene,9,12,21,MLMT performance score improvement at 1 year,1
HLT011-000056,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,0,24,24,Androstenedione normalization at 24 weeks,1
HLT011-001148,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,8,24,32,Androstenedione normalization at 24 weeks,1
HLT011-000178,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,3,24,27,Androstenedione normalization at 24 weeks,1
HLT011-000001,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,0,24,24,Androstenedione normalization at 24 weeks,1
HLT011-000529,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,1,24,25,Androstenedione normalization at 24 weeks,1
HLT011-001118,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,4,24,28,Androstenedione normalization at 24 weeks,1
HLT011-000403,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000185,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,2,24,26,Androstenedione normalization at 24 weeks,1
HLT011-001416,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,0,24,24,Androstenedione normalization at 24 weeks,1
HLT011-001160,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,9,24,33,Androstenedione normalization at 24 weeks,1
HLT011-000495,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,9,24,33,Androstenedione normalization at 24 weeks,1
HLT011-000662,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,9,24,33,Androstenedione normalization at 24 weeks,1
HLT011-000842,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,10,24,34,Androstenedione normalization at 24 weeks,1
HLT011-000296,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000152,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000345,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000073,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000459,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,6,24,30,Androstenedione normalization at 24 weeks,1
HLT011-000993,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,8,24,32,Androstenedione normalization at 24 weeks,1
HLT011-001324,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,5,24,29,Androstenedione normalization at 24 weeks,1
HLT011-000627,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,9,24,33,Androstenedione normalization at 24 weeks,1
HLT011-000639,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,11,24,35,Androstenedione normalization at 24 weeks,1
HLT011-000242,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,10,24,34,Androstenedione normalization at 24 weeks,1
HLT011-000708,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,0,24,24,Androstenedione normalization at 24 weeks,1
HLT011-000715,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Placebo,0,24,24,Androstenedione normalization at 24 weeks,1
HLT011-000426,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,6,24,30,Androstenedione normalization at 24 weeks,1
HLT011-000105,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,8,24,32,Androstenedione normalization at 24 weeks,1
HLT011-001074,TRIAL-CAH-001,Congenital Adrenal Hyperplasia,Rare endocrine,Crinecerfont,5,24,29,Androstenedione normalization at 24 weeks,1
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