HEMOPHILIA A AND B
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Hemgenix 4-year results show steady durability
MONTREAL, March 12, 2025 – Results from the HOPE-B trial for the hemophilia B gene therapy, Hemgenix, show steady expression of factor IX (FIX) four...
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Development halts in first-generation gene therapies
MONTREAL, February 25, 2025 – On February 20, Pfizer announced that it was stopping development of its hemophilia B gene therapy, Beqvez. It cited “soft...
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Novel treatments in bleeding disorders: The European Haemophilia Consortium Review
CLICK HERE to access the 2025 ISSUE ONE of the periodic review of novel treatments in hemophilia and other bleeding disorders by the European Haemophilia...
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CHS survey of people with hemophilia A and their caregivers
In June and July 2024, the Canadian Hemophilia Society conducted an online survey regarding the lived experience of people with hemophilia A and their caregivers....
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Biomarin limits marketing of its FVIII gene therapy, Roctavian
SAN RAFAEL, California, August 5, 2024 — Biomarin has announced that it will limit commercial, research and manufacturing programs for its factor VIII gene therapy...