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.

At this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving ...

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

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.

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.