Gene therapy trial offers hope for boys with devastating muscle disease
Disease control
Recruiting now
This study is testing an experimental gene therapy called SRP-9001 (delandistrogene moxeparvovec) in boys with Duchenne muscular dystrophy (DMD). The main goals are to see if the one-time treatment is safe and if it helps the body produce a crucial muscle protein called dystrophi…
Phase: PHASE1 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC