One-Shot gene therapy tested to fight devastating muscle disease in boys
Disease control
Completed
This early-stage trial tested a one-time gene therapy injection for boys with a specific genetic form of Duchenne muscular dystrophy. The goal was to see if the treatment was safe and could help their muscles produce a functional version of a crucial protein called dystrophin. Th…
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC