On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...

Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...

Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...

Oliver Chu has shown “dramatic improvements” following the groundbreaking treatment, his father Ricky Chu said ...

Harlow is defying the odds after receiving groundbreaking gene therapy for a rare brain disorder. Her progress has been called "a miracle" by her family ...

Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

NEW DELHI, Dec ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...