PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

EDO51 for Duchenne muscular dystrophy after Phase 2 trial failure, abandons three preclinical programs, and pivots to focus ...

A rare and fatal form of muscular dystrophy has long posed a devastating diagnosis for children, primarily affecting boys.

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

CureDuchenne, a global nonprofit based in Newport Beach and dedicated to finding a cure for Duchenne muscular dystrophy, will ...