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 ...

Intellia's news comes at a challenging time for gene therapies, as multiple serious adverse events and fatalities have been ...

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

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

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 ...

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 ...

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

Cell and gene therapy (C&GT) is set to revolutionise treatment of complex diseases, offering new possibilities for patients.

The death of a patient in a trial of an experimental gene therapy casts new shadows over a technology once seen as the future ...

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

Last year, Jace was diagnosed with Duchenne muscular dystrophy ... chief research officer of the Muscular Dystrophy Association. Elevidys works by delivering a shortened dystrophin gene, called ...