duchenne muscular dystrophy gene therapy

News

FDA Will Ask Sarepta to Halt Gene Therapy Shipments

STAT · 2h

In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

Devdiscourse · 1h

Sarepta Suspends Gene Therapy Shipments After Tragic Deaths

Sarepta Therapeutics has paused U.S. shipments of its gene therapy Elevidys after two teenagers with Duchenne muscular dystrophy died of liver failure. The suspension follows an FDA request and affects non-ambulatory patients.

Reuters on MSN · 1h

Sarepta to pause Elevidys gene therapy shipments in US

Sarepta Therapeutics said on Monday it will pause all shipments of Elevidys in the United States after two teenage boys with a rare condition called Duchenne muscular dystrophy, who had received the company's gene therapy,

MedPage Today10h

Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

1don MSN

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Zacks Investment Research on MSN11h

SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...

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 ...

Fierce Biotech11h

Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...

STAT3dOpinion

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 ...

Muscular Dystrophy News4d

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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