Research and Clinical Trials

If you are interested in receiving information about clinical trials available for Duchenne Muscular Dystrophy please email Dr. Ciafaloni.

Current Clinical Trials

 

1. A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intratechal Injection of INS1201 in Ambulatory Males with Duchenne Muscular Dystrophy (The ASCEND study). Target Population: Males, 2 to < 5years of age with Duchenne Muscular Dystrophy
2. A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION). SRP-9001-303
3. A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study (EXPEDITION)
4. A Phase 2 Randomized, Double-blind, placebo-controlled study to evaluate the effect of EDG-5506 on Safety, biomarkers, Pharmacokinetics, and functional measures in Adults and adolescents with Becker Muscular Dystrophy (GRAND-CANYON)
5. The Baby Duchenne Study: Characterizing developmental and clinical outcomes in the first three years in children with Duchenne Muscular Dystrophy.