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) ...

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 ...

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 ...

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 ...

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 ...

Edgewise Therapeutics Inc. (Nasdaq: EWTX) has initiated a study of the impact of its flagship muscular dystrophy drug candidate EDG-5506 on children with Duchenne muscular dystrophy. The Phase 2 ...

Duchenne Muscular Dystrophy is a genetically inherited disorder that causes muscles to progressively weaken and waste. It is X-linked, where sons inherit it from their mothers, or in rare occasions, a ...

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester ...

A collaboration between UT Southwestern and Children’s Health has been recognized for its work toward finding a cure for Duchenne muscular dystrophy. The program is now a Certified Duchenne Care ...

Aw! A 6-year-old boy with Duchenne muscular dystrophy recently popped the question to his art teacher at Duke Children's Hospital. Creed, who is from the Fayetteville area, has been treated at Duke ...

WILMINGTON, N.C. (WECT) -New Hanover County Fire Rescue firefighters are collecting donations at intersections around ...