A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy
rAAVrh74.MHCK7.micro-dystrophin (SRP-9001) was designed to safely express a functional micro-dystrophin protein in skeletal and cardiac muscle. The purpose of Study 102 is to evaluate the safety and efficacy of IV SRP-9001 in people with DMD by measuring biological and clinical endpoints in a Phase 2 multicenter, randomized, double-blind, placebo-controlled trial (NCT03769116). Results from the 41 patients that have been randomized and dosed (SRP-9001 or placebo) in Part 1 will be presented.