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

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 ...

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 ...

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

Share this page with email Share this page on Facebook Share this page on X Share this page on Reddit Print Preferred Source on Google Muscular Dystrophy News Today is wrapping up coverage of the 2026 ...

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