Bridgebio Pharma asked the FDA to approve BBP-418, an oral therapy that could be the first treatment for LGMD2i.

Columnist Robin Stemple's latest excursion was a trek across the city to discover where to buy a lift chair for his ...

Sarepta plans to ask the FDA for full approval of exon-skipping Duchenne muscular dystrophy treatments Amondys 45 and Vyondys 53.

Early trial data suggest RNA therapies SRP-1001 and SRP-1003 reach muscle and show biomarker activity in muscular dystrophy.

Dyne Therapeutics is advancing z-rostudirsen toward approval for DMD after data showed increased dystrophin and early ...

The evidence for Elevidys is growing, and Sarepta is working to make the DMD treatment widely available, the company's ...

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of ...

The MDA's annual conference highlighted recent breakthroughs and the outlook for future care in the neuromuscular disease community.

Columnist Betty Vertin says her three sons with DMD may not have a big social life, but they have a big family that makes up ...

Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...

Dyne Therapeutics' Z-basivarsen led to gains in motor function and cognition for myotonic dystrophy type 1, and is entering a Phase 3 trial.