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Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy

key information

study id #: NCT03375164

condition: Duchenne Muscular Dystrophy

status: active, not recruiting


The proposed clinical trial is a single-dose controlled trial using rAAVrh74.MHCK7.micro-dystrophin for DMD patients. Cohort A will include six patients ages 3 months to 3 years, and Cohort B will include six patients ages 4 to 7 years old. All patients will receive intravenous SRP-9001.

intervention: rAAVrh74.MHCK7.micro-dystrophin

mechanism of action: Gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT03375164

last updated: November 14, 2020