duchenne muscular dystrophy gene therapy

News

Sarepta, Duchenne and gene therapy

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.

Yahoo Finance · 17h

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.

EconoTimes · 6h

FDA Probes Death of 8-Year-Old Linked to Sarepta’s Elevidys Gene Therapy

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta Therapeutics (NASDAQ: SRPT) gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD).

MIT Technology Review22h

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.

STAT20h

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 ...

20h

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

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

8hon MSN

FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy

The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...

Medscape16h

EMA Says No to Duchenne Gene TherapyElevidys

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

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 ...

10hon 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 ...

Fierce Biotech4d

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 ...

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