Fox News

Muscular dystrophy breakthrough: FDA approves first-ever gene therapy for rare children’s disease

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 ...

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community

The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed by Novartis, a major step forward for ...
kcra.com

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids

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 ...
WVXU

Muscular dystrophy may be able to be prevented, Cincinnati Children's researchers say

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) ...
Morningstar

Parent Project Muscular Dystrophy Announces Children's Hospital of Philadelphia as Certified Duchenne Care Center, Recipient of Inaugural Clinical Research Designation

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 ...