THERAPIES
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Best Medicines Coalition submission on high-cost drugs for rare diseases
The Best Medicines Coalition (BMC) is a national alliance of patient organizations, together representing millions of patients, with a shared goal of equitable, timely and...
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CHS replies to Quebec’s INESSS following its shocking report on the funding of Hemlibra
The Institut national d’excellence en santé et en services sociaux (INESSS) released its recommendation on funding Hemlibra (emicizumab) for the treatment of severe hemophilia A...
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Fitusiran trial restarts with new controls
BRUSSELS – During the European Association for Haemophilia and Allied Disorders (EAHAD) virtual congress session on the latest clinical trial, Dr. Shauna Anderson, representing Sanofi,...
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Community awaits decisions on access to Hemlibra
Montreal, February 4, 2021 – The hemophilia community is awaiting decisions by Canadian health authorities on access to Hemlibra for people with hemophilia A without...
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FDA puts uniQure’s gene therapy clinical programme, HOPE-B, on hold following submission of a safety report
December 21, 2020 – The European Haemophilia Consortium (EHC) has learned that uniQure announced that its haemophilia B gene therapy programme, including the pivotal, phase...