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 ...

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