There are currently no available treatment options for more than 90% of the approximately 7,000 rare diseases identified to ...

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

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

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

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the life-threatening bleeds caused by the condition.

A groundbreaking gene therapy has successfully restored immune function in children with ADA-SCID, a severe genetic disorder.

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

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

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

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

Miracles happen every day, everywhere, in scenarios we can hardly imagine.Although human life has become so complex that we ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...