Genetic Disease, Inborn
5
3
3
2
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
0.0%
0 terminated out of 5 trials
100.0%
+13.4% vs benchmark
60%
3 trials in Phase 3/4
50%
1 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 2 completed trials
Clinical Trials (5)
Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
A Study Comparing the Quality of Life of Patients in the Treatment of Eczema by Pediatric Generalists and Specialists