Update Long-Term Follow-Up In Babies Born With ‘Bubble Boy Disease’ Still Seem Cured

Update Long-Term Follow-Up In Babies Born With ‘Bubble Boy Disease’ Still Seem Cured

Update Long-Term Follow-Up In Babies Born With ‘Bubble Boy Disease’ Still Seem Cured 6 of the children treated for SCID living their best lives – credit, UCLA, supplied by the parents Between 2012 and 2017, 62 babies and toddlers were treated with a genetic therapy for severe combined immunodeficiency, known colloquially as the “Bubble Boy disease.” In 2021, GNN reported on the results of the trial—that by 2019, 95%, or all but two of the young patients, showed complete immune system reconstruction. Now, the long-term follow-up results are in—still 95% effective. Screenshot, GNN “The durability of immune function, the consistency over time and the continued safety profile are all incredibly encouraging,” said the study’s senior author, Donald Kohn, MD, a pediatric transplant physician at Univ. of California LA, where the trial was conducted. Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in the ADA gene, which creates an enzyme es…