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.

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

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

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

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

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 ...

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

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 ...

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

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.