News

Roche pauses shipments of Elevidys gene therapy outside US
Roche Holding AG said on Tuesday it has paused all shipments of muscular disorder gene therapy Elevidys outside the U.S., ...
Roche Pauses Shipments of Contested Gene Therapy Outside the US
Roche Holding AG paused all shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...
Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that ...
Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy
Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...
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 Today1d
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 ...
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 ...
MedPage Today on MSN5d
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 ...
EMBO Press1h
miR-33 inhibition as a novel therapeutic approach for treating muscular dystrophy
MicroRNA-33a and microRNA-33b are encoded within the mammalian SREBF1 and SRBF2 genes, respectively, and both SREBF1 and ...
Zacks.com on MSN1d
SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program
Sarepta plummets after a third death in its muscular dystrophy program for investigational gene therapies, prompts FDA action and intensifies safety scrutiny.
Devdiscourse5h
Roche Pauses Global Shipments of Elevidys Gene Therapy
Roche Holding AG temporarily halted shipments of its muscular disorder gene therapy, Elevidys, outside the U.S., mirroring its U.S. partner Sarepta Therapeutics. The pause affects orders relying on ...
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 ...