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 ...

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