Intellia’s stock fell 23% after one patient in its Phase III MAGNITUDE trial developed severe liver toxicity that later ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

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

CureDuchenne, a global nonprofit based in Newport Beach committed to finding and funding a cure for Duchenne muscular ...

Boston-based biotech PepGen Inc. (NASDAQ:PEPG) announced today it will discontinue all Duchenne muscular dystrophy (DMD) ...

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

Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a ...

Living with a rare form of muscular dystrophy, and being honoured with having the youngest-ever seat on the board of trustees ...

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

Families began receiving calls from doctors last week to arrange treatments for new Givinostat drug after a campaign by the ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

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