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

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

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 ...

MDA 2026 keynote speaker John Crowley discussed with MD News Today the progress made in neuromuscular disease and the future ...

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

Finding knowledgeable, expert clinicians to treat rare conditions is a challenge in itself, says guest writer Kelly Berger, who offers tips.

Because technology is a part of columnist Shalom Lim's daily life with Duchenne muscular dystrophy, he weighs in on the ...

Columnist Patrick Moeschen recalls how he worried about falling behind classmates in the years before his MD diagnosis.

People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.