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 ...

The FDA will submit a boxed warning and other labeling limits for Elevidys, an adeno-associated virus and vector-based gene ...

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) ...

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, ...

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 ...

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 ...

At the cellular level, the mechanics of how muscle tissue repair occurs gets complicated. There are significant differences ...

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 ...

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