gene therapy duchenne muscular dystrophy

News

FDA Will Ask Sarepta to Halt Gene Therapy Shipments

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

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The Massachusetts-based biotech company is under fire for refusing a request from the U.

Associated Press News on MSN · 17h

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

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene therapy.

Southwest Journal · 1d

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

Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% as company halts non-ambulatory patient treatments.

STAT1d

Patient dies in Sarepta gene therapy trial, adding to safety concerns

A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...

MedPage Today on MSN2d

Duchenne Gene Therapy Will Undergo Changes After Patient Deaths

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

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 ...

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 ...

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 ...

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 ...

STAT1d

Sarepta Therapeutics crisis is huge blow to Duchenne families, company

The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, ...

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 ...

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