资讯

STAT1 小时Opinion
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 ...
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 ...
MedPage Today1 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 ...
Zacks Investment Research on MSN2 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 ...
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 the FDA.
Fierce Pharma3 小时
Sarepta stands behind Elevidys after FDA requests gene therapy be pulled from market
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm ...
Novel approach enhances neuromuscular function in patients with Duchenne muscular dystrophy
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...
Sarepta Therapeutics stock sinks after FDA tells company to stop shipping its bestselling drug
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an ...
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 ...
Precision Medicine Online2 小时
Sarepta to Continue to Ship Duchenne Gene Therapy Elevidys Despite FDA Request
The FDA requested Sarepta voluntarily stop distributing Elevidys after the death of a third patient who received one of the firm's gene therapies.
MyChesCo on MSN36 分钟
FDA Shuts Down Sarepta Gene Therapy Trials After Three Deaths Linked to Liver Failure
The U.S. Food and Drug Administration on Friday placed Sarepta Therapeutics’ gene therapy trials for limb girdle muscular ...
BioPharma Dive2 天
FDA to ask Sarepta to stop shipping Duchenne gene therapy
The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...