Muscular Dystrophy, Duchenne
47
6
8
30
Key Insights
Highlights
Success Rate
83% trial completion
Published Results
15 trials with published results (32%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
12.8%
6 terminated out of 47 trials
83.3%
-3.2% vs benchmark
23%
11 trials in Phase 3/4
50%
15 of 30 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 30 completed trials
Clinical Trials (47)
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Regular Physical Exercise in Duchenne Muscular Dystrophy
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
Characterization of DupEx2 Duchenne Muscular Dystrophy
Spironolactone Versus Prednisolone in DMD
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)