A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Dyne Therapeutics reports positive long-term data for DYNE-251 in Duchenne muscular dystrophy ... the Phase 1/2 ACHIEVE trial in myotonic dystrophy type 1 (DM1). DYNE-251 is an investigational ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Roche (RHHBY) announced that it will present new data at the Muscular Dystrophy Association, MDA conference, 16-19 March, 2025, in Dallas, ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy ...

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...