GENE THERAPY
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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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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,...