The investigational gene therapy SGT-003 is showing early signs of safety and efficacy for Duchenne muscular dystrophy in a ...
PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...
Columnist Robin Stemple knows that his physical challenges aren't his fault, but that doesn't make it any easier to avoid feeling ashamed.
People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.
MDA 2026 keynote speaker John Crowley discussed with MD News Today the progress made in neuromuscular disease and the future challenges.
Columnist Patrick Moeschen recalls how he worried about falling behind classmates in the years before his MD diagnosis.
Consuming more dietary protein may benefit people with various types of muscular dystrophy, according to a recent study.
Living with a rare disease is incredibly complex, with countless layers to navigate. Managing daily life amid so many unknowns — and trying to find a regimen that truly works — can feel overwhelming.
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