duchenne muscular dystrophy gene therapy

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FDA asks Sarepta to stop shipping gene therapy

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 · 1d

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.

Yahoo · 2d

FDA to ask Sarepta to stop shipping Duchenne gene therapy

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the therapy, Elevidys.

Sarepta Therapeutics crisis is huge blow to Duchenne families, company

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

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 ...

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 ...

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 ...

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 ...

BioSpace4d

Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

STAT4d

Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move

Sarepta Therapeutics announced it has laid off more than one-third of its workforce, a drastic cost-cutting move following ...

News-Medical.Net on MSN5d

New cellular entry pathway improves gene therapy outcomes

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...

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