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 ...
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 ...
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 ...
STAT8h
Sarepta Therapeutics’ Duchenne therapy faces ‘arduous’ path back to market, senior FDA official says
Sarepta Therapeutics faces an "arduous path” to try to get its gene therapy for Duchenne muscular dystrophy back onto the market, a senior FDA official told STAT.