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

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 ...

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

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

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 ...

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

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