Nature Medicine, Published online: 27 June 2025; doi:10.1038/s41591-025-03750-3
In the primary report of a phase 1b trial, adeno-associated virus gene therapy with fordadistrogene movaparvovec in patients with Duchenne muscular dystrophy was well tolerated in an ambulatory cohort, and mini-dystrophin expression was increased in the high-dose cohort.
