GENE THERAPY
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Global dosing hold in fitusiran trials initiated by Sanofi Genzyme to investigate new adverse events
A joint statement of the World Federation of Hemophilia (WFH), European Haemophilia Consortium (EHC) and National Hemophilia Foundation (NHF) November 6, 2020 – The WFH,...
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Products in the pipeline
It is an exciting time in the treatment of people with bleeding disorders with many coagulation products having been recently approved by Health Canada or...
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WFH publishes 3rd Edition of the Guidelines for the Management of Hemophilia
The World Federation of Hemophilia (WFH) has published the 3rd Edition of the Guidelines for the Management of Hemophilia, replacing the 2nd Edition published in...
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FDA denies approval of Biomarin’s FVIII gene therapy
In a decision that surprised many, the U.S. Food and Drug Administration (FDA) last week denied approval of Biomarin’s FVIII gene therapy, branded Roctavian. 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...