News

Children's Hospital LA pauses use of Sarepta gene therapy for all patients
Children's Hospital Los Angeles, citing recent U.S. Food and Drug Administration actions, on Monday said it has paused usage of Sarepta Therapeutics' gene therapy Elevidys in all patients with ...
Investor's Business Daily on MSN2h
Sarepta's 'Unprecedented Times' Continue As Company Rebukes The FDA
Sarepta stock took another dive Monday after the biotech company refused the FDA's request to voluntarily stop all shipments ...
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
U.S. regulators recently asked Sarepta Therapeutics to voluntarily halt shipments of its Elevidys gene therapy after a ...
Sarepta shares hit as Mizuho, Leerink warn on FDA rift, credibility risk
Investing.com -- Sarepta Therapeutics came under pressure after two brokerages slashed their price targets and downgraded the stock, flagging mounting safety, regulatory, and credibility concerns ...
STAT4hOpinion
The crisis over Sarepta’s Duchenne therapy is a mess. But the lesson is clear
The crisis over Sarepta Therapeutics' Duchenne therapy offers a valuable lesson: Listening to critics, and responding with ...
MedPage Today5h
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 ...
BioPharma Dive5h
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 ...
News-Medical.Net5h
Gene editing in the brain offers hope for ultra-rare neurological disease
Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a ...
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 ...
University of California, San Francisco6h
Do These Two Cancer Drugs Have What It Takes to Beat Alzheimer’s?
Scientists at UCSF and Gladstone Institutes have identified cancer drugs that promise to reverse the changes that occur in ...
MPR6h
Third Death Prompts FDA Action on Sarepta’s Gene Therapy Products
The third death occurred in an adult male with limb girdle muscular dystrophy who received a Sarepta AAVrh74 gene therapy product, SRP-9004, in a phase 4 clinical trial.
News-Medical.Net6h
Combination of two cancer drugs could treat the most common form of dementia
A study comparing the gene expression signature of Alzheimer's disease with those elicited by 1,300 approved drugs found a combination of two cancer medications that could treat the most common form ...