Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

Roche Holding AG said on Tuesday it has paused shipments of muscular disorder gene therapy Elevidys in some countries outside the U.S., echoing a similar decision by U.S. partner Sarepta Therapeutics ...

The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received Sarepta Therapeutics' muscular disorder gene therapy Elevidys. The death ...

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...

With a strong launch underway for a bladder cancer gene therapy, Ferring is finding the kind of commercial success that’s ...

Sarepta Therapeutics said Friday that the deaths of two patients taking its marketed gene therapy Elevidys® (delandistrogene ...

Hansa Biosciences’ Idefirix (imlifidase) has reduced patients’ antibodies to a level that allows dosing in patients with ...

In March, Sarepta Therapeutics, Inc. (NASDAQ: SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.

Sarepta Therapeutics Inc. shares rose sharply early Tuesday after US regulators reversed course and recommended that patients who can walk be allowed to take its gene therapy Elevidys again.

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...

ELEVIDYS is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the ...