By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...

Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...

Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...

The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...

Hubble Therapeutics LLC a patient-focused biotechnology company developing a gene therapy to cure pediatric blindness, announces the successful ...