News

Drugmaker declines FDA request to pull treatment after patient deaths
By Zachary Stieber Contributing Writer Sarepta Therapeutics will not agree to a regulatory request to pause shipments of a ...
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy to ambulatory people but maintain a halt it implemented June 15 for ...
MedPage Today1h
Gene Therapy May Hold Better Outcomes for Congenital Deafness Than Cochlear Implant
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts ...
Sarepta Stock Sinks. How Its Crisis Went From Bad to Worse.
Sarepta stock has tanked. Most investors don’t see the drugmaker’s executive management as credible following the failure to disclose a patient death, a BMO survey suggests.
STAT2hOpinion
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 ...
Courthouse News Service3h
Cancer drugs show promise as potential Alzheimer’s treatment
Researchers say that a pair of already FDA-approved medications could reverse brain changes in the most common form of ...
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 ...
BioPharma Dive3h
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.Net3h
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 Francisco4h
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 ...
MPR4h
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.