The primary report of the EMBARK phase 3 trial, testing the AAV-based gene therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy, did not meet its primary endpoint of improvement in NSAA mobility scores compared to placebo. Secondary endpoints show that the therapy was safe and associated with improvements in micro-dystrophin expression and in individual mobility scores.
- Jerry R. Mendell
- Francesco Muntoni
- Louise R. Rodino-Klapac