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3-Year Outcomes of AAV Gene Therapy in Hemophilia B: Study Published in NEJM Shows Long-Term Factor IX Expression
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.

 

Hemophilia B

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Andrew M. Davidoff bleeding disorders Edward G.D. Tuddenham Gene therapy Headlines Hematology Hemophilia B Hemostasis Hemostasis Today Jenny McIntosh Malo Journou NEJM Pratima Chowdary Study Ulrike M. Reiss Vincent Muczynski
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