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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...
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OCTOSTIM SPRAY discontinued
Toronto, July 22, 2020 – Ferring Pharmaceuticals has discontinued production of OCTOSTIM SPRAY (150 mpg DIN 02237860) for the treatment of mild and moderate hemophilia A...
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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,...