DMD drug tested in wheelchair users – but trial stops early
Disease control
Terminated
This study tested the safety of golodirsen (Vyondys 53) in boys and men with Duchenne muscular dystrophy who can no longer walk. Only 2 people took part before the trial was stopped early. Participants received weekly IV infusions for up to 96 weeks, with extra follow-up. The goa…
Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC