A 13-year study by St. Jude Children’s Research Hospital and University College London demonstrates the long-term efficacy and safety of a one-time gene therapy for hemophilia B. Patients treated more than a decade ago maintain stable levels of clotting factor IX, significantly reducing their bleeding episodes without serious side effects. This landmark follow-up bolsters the promise of genetic therapies as lifelong treatments, especially for genetic disorders that historically required lifelong management.
Source: St. Jude / UCL (New England Journal of Medicine)
Link: https://www.stjude.org/media-resources/news-releases/2025-medicine-science-news/hemophilia-b-gene-therapy-demonstrates-long-term-success.html
3-Year Follow-Up Confirms Long-Term Safety of Hemophilia B Gene Therapy
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