gene therapy duchenne muscular dystrophy

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Duchenne, European Medicines Agency and gene therapy

Sarepta woes mount as Duchenne gene therapy knocked back in Europe

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could further jeopardize Sarepta’s financial future.

· 2h

Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU

STAT · 7h

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

· 1d

InnovationRx: Sarepta Blinks In Showdown With FDA

The latest twist in the saga followed a decision by Children’s Hospital Los Angeles to halt use of the drug while it was facing regulatory uncertainty.

· 2d

Sarepta falls after gene therapy maker pauses Elevidys shipments in US

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Sarepta pares gains as report suggests an ‘arduous’ path for Duchenne drug

STAT1h

Europe moves to reject embattled Duchenne muscular dystrophy gene therapy

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing scrutiny in the U.S.

MIT Technology Review4h

The deadly saga of the controversial gene therapy Elevidys

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

1hon MSN

Sarepta fails to win EU backing for muscle disorder gene therapy

Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the approval of Elevidys, following mounting regulatory scrutiny and two recent patient deaths linked to the company's gene therapy.

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 ...

MedCity News2y

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

The Duchenne muscular dystrophy drugs now available only treat the symptoms of the rare muscle-wasting disorder. On Thursday, the FDA approved a Sarepta Therapeutics gene therapy, making it the ...

4don 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 gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.