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Two deaths in gene therapy trial for rare neuromuscular disease
The CHS recently learned of two deaths of children under five years old in a clinical trial using AAV8-based vectors in another rare disease area,...
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Four-year follow-up of Biomarin’s FVIII gene therapy
Dr. John Pasi, Centre Director at the Royal London Haemophilia Centre, presented 4-year follow-up data on Biomarin Pharmaceutical’s Phase 1 /2 gene therapy trial of...
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Redefining prophylaxis
A series of renowned speakers at the World Federation of Hemophilia Virtual Summit re-defined prophylaxis in this era of innovative therapies, including non-factor replacement therapies....
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CHS Submission to the Canadian Agency for Drugs and Technologies in Health (CADTH) on Hemlibra
Montreal, July 21, 2020 – The Canadian Hemophilia Society has in recent days made submissions to both CADTH (Canadian Agency for Drugs and Technologies in...
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Dangerously high FIX levels reported in hemophilia B gene therapy
Montreal – June 29, 2020 – During the question-and-answer period of a session on gene therapy during the World Federation of Hemophilia’s virtual summit (June...