“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...
New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...
Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...
Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...
Results that may be inaccessible to you are currently showing.
Hide inaccessible results
Feedback