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Gene Therapy Stays on Market Despite FDA Request
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A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...
Zacks Investment Research on MSN1h
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 ...
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...
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 ...
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...
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