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Medical Daily
Medical Daily

Gene Therapy for Children With Rare 'Bubble Boy Disease' Proves Effective After Long-Term Follow-Up

Children who were found to have the rare "Bubble Boy disease" as babies and toddlers still seem cured after long-term follow-up after being treated with gene therapy.

The 62 children who were found to have the rare "Bubble Boy disease" as babies and toddlers between 2012 and 2017 still seem cured after long-term follow-up, after being treated with a genetic therapy for severe combined immunodeficiency.

The results of the trial noted that by 2019, 95 percent of the children, which equates to all but two of the young patients, showed complete immune system reconstruction. And now, years later, long-term follow-up results show that the therapy is still 95 percent effective.

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