The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

WASHINGTON, June 12, 2025 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is pleased to announce the ...

In the spirit of the holidays, we're looking back on the time the Grateful Dead played a surprise set for kids with muscular ...