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 ...

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 ...

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.

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 ...