Jun 13, 2025, 07:54
3-Year Outcomes of AAV Gene Therapy in Hemophilia B: Study Published in NEJM Shows Long-Term Factor IX Expression
The New England Journal of Medicine posted on X:
“Liver-targeting adeno-associated virus–mediated gene therapy led to stable long-term factor IX expression and a decrease in bleeding episodes for up to 13 years in 10 men with hemophilia B. Full study results.”
Title: Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
Authors: Ulrike M. Reiss, Andrew M. Davidoff, Edward G.D. Tuddenham, Pratima Chowdary, Jenny McIntosh, Vincent Muczynski, Malo Journou et al.
Check the full article here.
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