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

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

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

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

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

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

A uniQure gene therapy slowed progression of Huntington’s disease by 75% after three years, statistically significant clinical trial results the company says will support plans for a regulatory ...

A gene therapy that targets a form of retinitis pigmentosa showed a signal to improve retinal function for up to 3 years after treatment, according to a small clinical trial. The phase 2 SKYLINE trial ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...