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

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

From a missing sign turning up across the Irish sea, to cancelled festivals and teenagers locked in a play park - here are ...

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

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

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

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

Taevion Norris was forced to leave his classmates at West Leyden High School in Northlake after being diagnosed with heart ...