duchenne muscular dystrophy

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Sarepta's Duchenne Therapy Sparked Fears

Roche halts some shipments of Sarepta’s Duchenne gene therapy outside U.S.

Roche is following its partner Sarepta Therapeutics and stopping shipments of the Duchenne muscular dystrophy gene therapy Elevidys in some countries, amid rising safety concerns surrounding the trea...

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Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy

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Sarepta’s Duchenne Therapy Sparked Fears Even Before FDA’s Order

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Sarepta Faces Fresh Setback. Why Now’s Not the Time to Buy the Stock.

Sarepta Therapeutics was falling again in premarket trading Tuesday as its future looks increasingly shaky after it agreed to halt shipments of its Elevidys drug under pressure from the Food and Drug ...

STAT · 1d

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

STAT · 1d

Pharmalittle: We’re reading about Sarepta resisting FDA over Elevidys, agency naming top drug regulator, and more

BioSpace1h

Following Sarepta’s Lead, Roche Suspends Elevidys Shipments to Some Ex-US Countries

Discovered and developed by Sarepta Therapeutics, Elevidys is a gene therapy that won accelerated approval in June 2023 and full approval in June 2024. Roche partnered with Sarepta in December 2019, paying $1.15 billion upfront in cash and stock for the right to launch and market Elevidys outside the U.S.

The BMJ2h

Duchenne gene therapy halted after three deaths and company u turn

The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and Drug Administration (FDA) to pull the drug from the market.

Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need,

6hon MSN

Roche Pauses Contested Gene Therapy’s Shipments Outside US

Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled US biotechnology company that developed the treatment.

2don 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.

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Food and Drug Administration

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 dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.

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

Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, according to a STAT report citing a senior FDA official, suggesting the treatment’s license could be revoked amid ongoing safety concerns.

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 never asked to join — said goodbye to their sons, the babies they once held in their arms, whose dreams they held in their hearts until Duchenne robbed them of working muscles or a healthy future.