Medical and Scientific News
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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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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...