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

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

Contributed by Louis M. Kunkel, April 18, 2017 (sent for review March 6, 2017; reviewed by Jeffrey S. Chamberlain and Melissa J. Spencer) ...

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

Timing of the surgery varies and may be dictated by the underlying disorder. In the presence of muscular dystrophy, for example, we consider surgical intervention once the child is wheelchair-bound, ...

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 ...

PepGen Inc. (NASDAQ:PEPG) expects data from its Phase 1 FREEDOM-DM1 trial’s 15 mg/kg cohort in the second half of 2025, and ...

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 ...

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

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