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hemophilia B

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Posted inHematology-Oncology Internal Medicine news

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

Posted by MedXY By MedXY 12/19/2025
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 prophylaxis in the vast majority of participants.
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Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results
Posted inClinical Updates Hematology-Oncology news Specialties

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

Posted by MedXY By MedXY 08/01/2025
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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