资讯

Sarepta to pause Elevidys gene therapy shipments in U.S.
Sarepta Therapeutics (SRPT) said late Monday it will pause all shipments of its Duchenne muscular dystrophy gene therapy, ...
MedPage Today15 小时
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 ...
Devdiscourse6 小时
Sarepta Suspends Gene Therapy Shipments After Tragic Deaths
Sarepta Therapeutics has paused U.S. shipments of its gene therapy Elevidys after two teenagers with Duchenne muscular ...
STAT7 小时
In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene ...
Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.
BioPharma Dive12 小时
FDA asks Sarepta to stop shipping Duchenne gene therapy
The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...
Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request
When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...
Zacks Investment Research on MSN16 小时
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 ...
Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
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 ...
BioPharma Dive15 小时
5 questions on Sarepta, the FDA and a Duchenne gene therapy crisis
A standoff over Elevidys could have major consequences for Duchenne patients, gene therapy companies and the perception of ...
USA Today2 年
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 ...
Nature20 年
Gene Therapy: The ‘pro-sense’ approach to Duchenne muscular dystrophy
After years of struggle to develop a gene therapy for Duchenne muscular dystrophy (DMD), there now seems to be a tool with which we can utilize an escape route that nature had already hinted at.