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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
The Phase 3 EMBARK trial investigates AAV gene therapy (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). The study assessed improvements in motor function, micro-dystrophin expression, and timed motor tests over 52 weeks. While the therapy showed some positive results, the primary outcome (North Star Ambulatory Assessment) did not reach statistical significance. Most side effects were mild, with a few serious cases resolved without fatal outcomes. The findings suggest AAV gene therapy is promising but requires further validation in future trials.
