For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

The trial was small — just 12 children were treated in one or both ears — but it offers important lessons for researchers ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

Opal Sandy, now 3, underwent a gene therapy injection to her right ear in 2023. Top admiral steps down abruptly amid drug ...

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

ICH gives CGT developers a regulatory framework that finally matches the speed and flexibility their groundbreaking therapies ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of ...