Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for a rare muscle disorder.

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy ...

Learn more about the generative AI technology that will be used at the Food and Drug Administration (FDA) to speed up reviews ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

Since the approval of Luxturna, there has been a wave of gene therapies advancing in clinical trials to treat genetic vision ...