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

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

Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung ...

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

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

Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...

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

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

Rob Stemple is a lifelong advocate for people with disabilities. He was diagnosed with FSHD in 1971 at age 14. Rob struggled with its’ affects for over 50 years. He lost his eyesight in a devastating ...

Columnist Robin Stemple was thrilled to see fellow FSHDers' creations during a virtual art show hosted by the FSHD Society wellness group.

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