Hayden Yung finds ways to persevere with muscular dystrophy, but experts say targeted help is needed both for children and ...

FIRDAPSE boosts Catalyst's strong Q1 2025 results, beating forecasts. With 15–20% growth projected, see why analysts rate ...

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 ...

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.

A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.