Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...

Use of DYNE-101 is leading to improvements in muscle, and specifically finger, function in myotonic dystrophy type 1 patients ...

Columnist Patrick Moeschen believes it's important to talk about what makes life fulfilling. For him, it's been a life of ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

Columnist Shalom Lim is sad to say goodbye to his caregiver of 18 years, but looks forward to the new chapter of life this ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

An onslaught of new setbacks due to FSHD disease progression has left columnist Robin Stemple understandably anxious about ...

Columnist Betty Vertin says her youngest daughter is starting to become aware of her older brothers' health challenges with DMD.

Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males with Duchenne muscular dystrophy (DMD), according to data from the ...

Columnist Betty Vertin has a day of heightened awareness about her sons' experience with wheelchairs and life with DMD.