THE BLOOD FACTOR
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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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Dangerously high FIX levels reported in hemophilia B gene therapy
Montreal – June 29, 2020 – During the question-and-answer period of a session on gene therapy during the World Federation of Hemophilia’s virtual summit (June...
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Biomarin’s gene therapy for hemophilia A to be called Roctavian
Montreal, May 14, 2020 – Biomarin announced on May 11 its gene therapy currently awaiting approval by the U.S. Food and Drug Administration will be...
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Gene Therapy Updates
FDA grants priority review for Biomarin’s hemophilia A gene therapy San Rafael, California, February 21, 2020 – Biomarin Pharmaceuticals has announced that the U.S. Food...
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No emicizumab this year for non-inhibitor patients
Montreal, April 17, 2020 – The Canadian Hemophilia Society (CHS) has learned that emicizumab (Hemlibra) will not become available for those with hemophilia A without...