But often the cancer will return and the entire bladder will be removed. “That comes like all kind of the side effects a ...

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

A patient died from liver injury while taking the gene therapy Elevidys Sarepta says more than 800 patients have received the ...

Scientists developed a promising gene replacement therapy for Dravet syndrome in mice. The therapy alleviated symptoms ...

NY resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, curing his sickle ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Gene therapy, the idea of fixing faulty genes ... activity and is small enough to be packaged within a non-pathogenic virus that can deliver ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without side effects.