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:...
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,...
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...
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....
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...
