New hope for boys with muscular dystrophy as major drug trial completes
Disease control
Completed
This large, completed study tested whether two drugs, casimersen and golodirsen, could help boys with a specific genetic form of Duchenne muscular dystrophy. For the first 96 weeks, some participants received the drug while others received a placebo, followed by a period where ev…
Phase: PHASE3 • Sponsor: Sarepta Therapeutics, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC