duchenne muscular dystrophy

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Sarepta's Duchenne Therapy Sparked Fears

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.

Bloomberg on MSN · 11h

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

Reports of troubles linked to Sarepta’s Elevidys treatment for Duchenne muscular dystrophy had been popping up on social media for months — beyond the two deaths the company had already disclosed — but there was little information from the company or the health regulators that widened availability of the drug over objections from its own staff,

Reuters on MSN · 4h

Roche pauses shipments of Elevidys gene therapy outside US

Roche Holding AG said on Tuesday it has paused all shipments of muscular disorder gene therapy Elevidys outside the U.S., echoing a similar decision by U.S. partner Sarepta Therapeutics on Tuesday.

FDA suspends muscular dystrophy drug

Newsweek on MSN · 1d

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.

Zacks Investment Research on M… · 1d

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 experimental gene therapies. The company confirmed that a 51-year-old, non-ambulant (unable to walk) limb-girdle muscular dystrophy (LGMD) patient died due to acute liver failure (ALF).

InvestorsHub on MSN · 1d

Sarepta Shares Drop as FDA Suspends Gene Therapy Trials for Muscular Dystrophy

Shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) plunged 10% after the company disclosed that the U.S. Food and Drug Administration (FDA) has imposed a clinical hold on several of its gene therapy programs targeting limb girdle muscular dystrophy (LGMD).

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 ...

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 ...

LEX 181h

Lemonade stand raising awareness and funds for rare genetic disease

A lemonade stand is a fairly common sight for a hot summer day in July. Outside of Kitchen Concepts in Lexington, a white and yellow stand is doing more than just satisfying thirst.

5hon MSN

Roche Pauses Shipments of Contested Gene Therapy Outside the US

Roche Holding AG paused all shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...

6hon MSN

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, ...

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Sarepta's Duchenne Therapy Sparked Fears

FDA suspends muscular dystrophy drug

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