Muscular Dystrophy, Duchenne
47
6
8
29
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
82.9%
-3.6% vs benchmark
21%
10 trials in Phase 3/4
52%
15 of 29 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 29 completed trials
Clinical Trials (47)
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Long-Term Outcomes of Ataluren in 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)
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)
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
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
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
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)