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

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

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 ...

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD ... Sarepta's gene therapy Elevidys – which is already ...

The death of a patient treated with Sarepta's Duchenne muscular dystrophy (DMD) gene therapy Elevidys has played havoc with its share price – although analysts believe that its benefits still ...

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

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

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

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

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

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

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