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Five-Year Durability of Etranacogene Dezaparvovec: Redefining the Long-term Management of Hemophilia B

By MedXY|Dec 19, 2025

The final 5-year analysis of the HOPE-B trial confirms that etranacogene dezaparvovec provides stable, long-term factor IX expression and clinical protection for patients with hemophilia B, effectively eliminating the need for routine proph

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results
Clinical UpdatesAAVclinical trial

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results

By MedXY|Sep 30, 2025

A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.

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