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.

Columnist Shalom Lim is using his lived experience with Duchenne muscular dystrophy to consult with organizations about ...

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

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

PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...

New presentations at MDA 2026 expand the understanding of how Duvyzat may help people with Duchenne muscular dystrophy.

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

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 ...

A human being since 1972, Patrick Moeschen recently retired after 28 years of teaching music at the middle school level in a public school setting. Diagnosed with Becker muscular dystrophy (MD) in ...

The investigational gene therapy SGT-003 is showing early signs of safety and efficacy for Duchenne muscular dystrophy in a ...