DMD drug safety study halted early after enrolling just 2 patients
Disease control
Terminated
This study looked at the safety of a drug called golodirsen in boys with Duchenne muscular dystrophy (DMD) who can no longer walk. The drug is given weekly through a vein and works by skipping a part of the genetic code to help produce a shorter but functional protein. Only 2 par…
Phase: PHASE4 • Sponsor: Rare Disease Research, LLC • Aim: Disease control
Last updated May 16, 2026 13:34 UTC