This early-stage study is testing a single dose of an experimental gene therapy called SGT-001 in boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and well-tolerated. Researchers will also check if the therapy helps produce a needed mu…
Phase: PHASE1, PHASE2 • Sponsor: Solid Biosciences Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC