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 ...

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) ...

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

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 ...

COLUMBUS, Ohio (WCMH) – It’s being called a pioneering event that holds great promise for children who have Duchenne muscular dystrophy. After decades of research, animal studies, and human trials, ...

A diagnosis of Duchenne muscular dystrophy (DMD) can come as a shock to parents and caregivers. Resources and support from family and friends can help you and your child navigate the diagnosis and ...

Karen Guillmeno’s son, Hunter, was diagnosed with Duchenne muscular dystrophy — a rare genetic disorder involving progressive muscle degeneration — in 2016. The condition primarily affects males in ...

Duchenne muscular dystrophy (DMD) results from changes in the DMD gene. Children may inherit DMD gene changes from their parents or have spontaneous gene changes. The DMD gene tells the body how to ...

Gene therapies have extended survival in muscular dystrophy, requiring improved care transitions from pediatric to adult systems. Current systems lack adequate staffing, specialists, and communication ...

HUDSON — Like many 20-year-olds, Jake Marrazzo struggles with time management and procrastination. So he figured he'd write a book about it, targeting younger children. "I feel like I've never really ...

FROM MUSCULAR DYSTROPHY. FOR WYATT ROWND, SPENDING THE DAY ON THE GOLF COURSE IS EXACTLY WHAT THE DOCTOR ORDERED. >> IT WAS AWESOME. GIANNA: HITTING GOLF BALLS HASN’T ALWAYS COME EASY TO WYATT. HE WAS ...

Some children in Northern Ireland who live with a severe form of muscular dystrophy will be able to access new medication on the health service, BBC News understands. The move from the Belfast Health ...