Sarepta Therapeutics (SRPT) said a third patient has died from acute liver failure after receiving one of its gene therapies, intensifying scrutiny over the safety of its treatments, Bloomberg ...

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 ...

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

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

AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant ...

Discover the current pipeline candidates advancing through the clinic for the treatment of sickle cell disease.

Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

The gene-therapy company is restructuring, laying off 36% of its workforce and making changes to its executive team.

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

CMS is brokering outcomes-based agreements on behalf of Medicaid programs. Thirty-three states, along with Washington, D.C., ...