DMD treatment KER-065 showed a good safety profile in a Phase 1 clinical trial, with no serious side effects seen in healthy ...

Treatment with gene therapy candidate BB-301 led to improvements in swallowing ability for the first three OPMD patients in a ...

I feel like the nutritionist is certainly overlooked by the patients, the families, as well as some teams. Families would decline a nutrition visit. And, more importantly, some insurance payers would ...

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

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

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 ...

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

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 ...

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

Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung function for boys with Duchenne muscular dystrophy (DMD), new analyses indicate.