Kids with 'bubble boy' disease could finally get access to a life-saving gene therapy, as a study starts back up at UCLA

Dec 27 2022 by Business Insider

 
Paola Andrea Fernández de Soto Abdul-Rahim holds her son, Jakob Kamil Guziak, inside their Edmonton house.

Kyler Zeleny

UCLA researchers are restarting a study to give a gene therapy to kids with an ultra-rare disease. While the one-time therapy is safe and effective, the drug industry has been uninterested.  The treatment is complex and costly, and the number of patients with SCID is tiny, limiting profits.

Researchers are now restarting a clinical trial to treat children with a severe...

Continue Reading Kids with 'bubble boy' disease could finally get access to a life-saving gene therapy, as a study starts back up at UCLA