News

MedPage Today on MSN3d
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
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 ...
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 ...
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 ...
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 ...
BioPharma Dive2d
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 ...
Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys
U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...
Exclusive: US FDA to ask Sarepta to stop shipments of gene therapy Elevidys, source says
The U.S. Food and Drug Administration is planning to ask Sarepta Therapeutics to voluntarily stop all shipments of its gene ...
STAT2d
Patient dies in Sarepta gene therapy trial, adding to safety concerns
A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...
STAT10d
FDA rejects Capricor’s cell therapy for Duchenne muscular dystrophy
The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...
Sarepta's Shocking Stand: Company Rejects FDA Request To Pull Gene Therapy Elevidys
FDA places clinical hold on Sarepta's LGMD gene therapy trials after three deaths and revokes platform tech designation due ...
Axios on MSN3h
Drugmaker refuses FDA request to pull gene therapy
An unusual public feud between the Food and Drug Administration and a maker of gene therapies for rare diseases could test ...