Duchenne Muscular Dystrophy
298
30
55
146
Key Insights
Highlights
Success Rate
79% trial completion
Published Results
66 trials with published results (22%)
Research Maturity
146 completed trials (49% of total)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
12.8%
38 terminated out of 298 trials
79.3%
-7.2% vs benchmark
12%
37 trials in Phase 3/4
45%
66 of 146 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 146 completed trials
Clinical Trials (298)
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Phase 2 Expanded Access Study of UC-MSC in DMD Patients
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
CureDuchenne Link®: A Resource for Research
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Parent and Infant Inter(X)Action Intervention (PIXI)
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
Early Check: Expanded Screening in Newborns
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort