This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any…
Phase: PHASE1, PHASE2 • Sponsor: Solid Biosciences Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC