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MedPage Today3h

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 ...
Zacks Investment Research on MSN5h

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 ...

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 ...
Fierce Biotech4h

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 ...

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 ...
STAT2dOpinion

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 ...
Muscular Dystrophy News4d

Dosing begins in gene therapy trial for DMD-linked heart disease

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...
BioSpace4d

Sarepta Tags DMD Gene Therapy Elevidys With Black Box Warning, Axes 500 Staff

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...