AAV Gene Therapy for Duchenne Muscular Dystrophy: The EMBARK Phase III Randomized Trial

A Phase III EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) was conducted . Ambulatory males with DMD, over four years to under eight years of age, were randomized and stratified by age group and North Star Ambulatory Assessment score to single-administration intravenous delandistrogene moxeparvovec or placebo.