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

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

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

Columnist Robin Stemple knows that his physical challenges aren't his fault, but that doesn't make it any easier to avoid ...

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 ...

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

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

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 ...

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

Capricor has reapplied for FDA approval of deramiocel, its therapy for DMD-related heart disease, and expects a decision by ...