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.

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.

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

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

Consuming more dietary protein may benefit people with various types of muscular dystrophy, according to a recent study.

The president and CEO of the Muscular Dystrophy Association talks about what she's most excited about in neuromuscular disease research.

A new 3D model shows that Duchenne gene therapy can repair muscle fibers but fails to shut down the signals that drive scarring.