gene therapy duchenne muscular dystrophy

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FDA asks Sarepta to stop shipping gene therapy

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· 4h

Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request

Facing mounting criticism, Sarepta recently announced plans to add a bold warning label to Elevidys.

· 21h · on MSN

Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths

Southwest Journal · 1d

Breaking: Second Patient Death Prompts FDA Investigation of Sarepta’s Elevidys Gene Therapy

· 20h

Sarepta tells FDA it won’t halt shipments despite patient deaths

Drugmaker Sarepta Therapeutics said late Friday it won’t comply with a request from the Food and Drug Administration to halt all shipments of its gene therapy following the death of a third patient r...

· 1d

FDA to ask Sarepta to stop shipping Duchenne gene therapy

· 1d

Sarepta Therapeutics crisis is huge blow to Duchenne families, company

USA Today2y

Duchenne muscular dystrophy treatments, gene therapy spark hope - USA TODAY

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Labroots2y

First Gene Therapy to Treat Duchenne Muscular Dystrophy is Approved

The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics Inc. Children aged four to five with the disorder and confirmed gene ...

MedCity News2y

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

The Duchenne muscular dystrophy drugs now available only treat the symptoms of the rare muscle-wasting disorder. On Thursday, the FDA approved a Sarepta Therapeutics gene therapy, making it the ...

People9mon

Boy with Duchenne's Gets Gene Therapy 3 Days Before He’s Ineligible ...

Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days before he ...

Science Daily5y

New gene correction therapy for Duchenne muscular dystrophy

New gene correction therapy for Duchenne muscular dystrophy. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2020 / 01 / 200127134851.htm

Muscular Dystrophy News2d

Dosing begins in gene therapy trial for DMD-linked heart disease

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated with Duchenne MD.