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 ...

RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing clinical trial, with no serious side effects reported. Interim data from the ...

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

Tomorrow evening, my longtime caregiver, Glenda, will leave her job after 18 years and 10 months of faithfully serving our family here in Singapore. She’ll be returning to the Philippines to be ...