Latest and Past News
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Hemophilia Research Million Dollar Club – Report from the chair
The Hemophilia Research Grants Review Committee, under the chairmanship of Dr. Manuel Carcao, met earlier this year and announced the 2020 grant recipients for the...
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Update of Phase 2b trial of UniQure’s AMT-061 gene therapy trial in hemophilia B
Dr. Steven Pipe, Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program in Ann Arbor Michigan, presented an update at the World Federation of...
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New hemophilia guidelines introduced at WFH Virtual Summit 2020
The global bleeding disorder community received an advance look at new international guidelines for the management of hemophilia at the WFH Virtual Summit 2020, at...
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Inhibitors – think differently
The development of inhibitory antibodies to therapeutic FVIII in up to 40% of severe hemophilia A (HA) patients is a major complication in the treatment...
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Musculoskeletal hot topic debates
In this session, three “hot topics” related to muscle, bone and joint health in people with bleeding disorders were debated by experts in their fields:...
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OCTOSTIM SPRAY discontinued
Toronto, July 22, 2020 – Ferring Pharmaceuticals has discontinued production of OCTOSTIM SPRAY (150 mpg DIN 02237860) for the treatment of mild and moderate hemophilia A...
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The WFH Gene Therapy and Bleeding Disorders Registries
WFH Gene Therapy Registry, presented by Dr. Barbara Konkle For many years gene therapy has been in the future – but now, for an increasing...
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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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My experience at the WFH Virtual Summit
I attended the WFH Virtual Summit and, although I would obviously have preferred to be there in person, I was impressed by the quality of...
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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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Critical need for harmonized post-marketing surveillance in gene therapy
With regulatory approval of the first gene therapy for clinical use outside of clinical trials anticipated in mid-2020, there is an urgent need to collect...
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Impressions of WFH Virtual Summit
Attending biennial World Federation of Hemophilia World Congresses was a highlight of my professional career in the pharmaceutical industry. From my first congress in Ireland,...