duchenne muscular dystrophy gene therapy

News

Roche pauses Sarepta gene therapy shipments outside U.S.

For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.

The Boston Globe · 2d

Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.

Yahoo · 6d

FDA asks Sarepta to stop shipping Duchenne gene therapy

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still walk. A pause on shipments to older, non-ambulatory patients remains in place.

MedPage Today3d

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 the agency was investigating the three deaths due to acute liver failure ...

Fierce Pharma7h

FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

Zacks Investment Research on MSN3d

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 ...

Medindia12mon

Gene Therapy Offers New Hope for Duchenne Muscular Dystrophy ... - Medindia

Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in ...

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 ...

STAT6dOpinion

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 ...

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