gene therapy duchenne muscular dystrophy

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FDA asks Sarepta to stop shipping gene therapy

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

At the heart of the controversy is a question many patients and families are asking: Should a drug that might save lives also be allowed to risk them?

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

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FDA to ask Sarepta to stop shipping Duchenne gene therapy

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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...

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Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says

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Sarepta Therapeutics crisis is huge blow to Duchenne families, company

STAT1dOpinion

We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we never asked to join — said goodbye to their sons, the babies they once held in their arms, whose dreams they held in their hearts until Duchenne robbed them of working muscles or a healthy future.

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 ...

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 News3d

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.