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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,...
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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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Redefining prophylaxis
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....