The Canadian Hemophilia Society's December 4, 2024 webinar on hemophilia gene therapy featured the latest research and access updates. Dr. Roy Khalifé described how AAV vector gene therapy works in hemophilia A and B. He reported the latest clinical trial results for of Roctavian (hemophilia A) and Hemgenix and Beqvez (hemophilia B), noting annualized bleeding rates and levels of factor VIII and IX expression. Safety concerns included liver enzyme elevations and the need for short-term corticosteroids. David Page addressed regulatory hurdles, noting Health Canada's approvals of Hemgenix and Beqvez, CDA's positive reimbursement recommendations, but INESSS' negative reimbursement recommendations for Quebec. The discussion emphasized the need for long-term monitoring and equitable access across Canada.

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In this podcast, Elizabeth Vansant, Mark Pervan, Murray Milne and Mathieu Jackson, all individuals with severe hemophilia B, are asked to debate the question: “If gene therapy for hemophilia B were available in Canada tomorrow, would you want to receive it?”

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In this podcast, Cleaven Pagani, Rick Waines, Paul Wilton and Emil Wijnker, all with severe hemophilia A and also members of the Canadian Hemophilia Society Blood Safety and Supply Committee, are asked to debate the question: “If gene therapy for hemophilia A were available in Canada tomorrow, would you want to receive it?”

See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.