GENE THERAPY
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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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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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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...