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 ...

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 functional trends.

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, ...

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

Capricor has reapplied for FDA approval of deramiocel, its therapy for DMD-related heart disease, and expects a decision by August 22.

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

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

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 ...