We’ve only had a mental health — I wouldn’t call it a crisis — but where mental health really became an issue. And it was with my oldest son, and he was still middle school age. So I feel like, as a ...

When living with Duchenne muscular dystrophy, taking a gamble feels better than not doing anything at all, columnist Betty Vertin writes.

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 ...

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 ...