At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was ...

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders ...

A third patient has died from acute liver failure after receiving a gene therapy treatment from Sarepta Therapeutics, the biotech company confirmed to Bloomberg on July 18. The patient died while ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...