Muscular Dystrophies
Pipeline by Development Stage
Drug Modality Breakdown
Competitive Landscape
7 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (10)
Total enrollment: 741 patients across 10 trials
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling
Ablelite Pediatric Device Clinical Study
Biomarker Development in LGMD2i
Phase Legend
Key Insights
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