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Bleeding Disorders

FDA Approves Hemophilia A Gene Transfer Rx

Today, July 3, 2023, the FDA approved the use of BioMarin’s Roctavian® [valoctocogene roxaparvovec] for treating adults with severe hemophilia A without inhibitors. The FDA also approved BioMarin’s companion diagnostic, AAV5 DetectCDx, which identifies the patients most likely to benefit from Roctavian. Click here for the LinkedIn announcement. Click here for a provocative FirstWord PHARMA […]
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Concizumab: Anti-TFPI

On 4-17-2023, Novo Nordisk announced Health Canada’s approval of Alhemo®, their trademark for injectable concizumab, the first anti-tissue factor pathway inhibitor [TFPI] designed to prevent bleeds in Hemophilia A and B patients with inhibitors. The current Canadian approval supports relatively few Hemophilia B [Christmas disease] patients with inhibitors but generalization to Hemophilia A patients could […]
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Webinar: The Many Faces of VWF Part 1: Von Willebrand Factor and Acquired von Willebrand Syndrome

Fritsma Factor participants, we invite you to view Part 1 of our video: series ‘Coag Conversations’  The Many Faces of von Willebrand Factor; or Von Willebrand Factor, ADAMTS13, Malignancy, and Beyond:  Von Willebrand Factor and Acquired von Willebrand syndrome, A Kaleidoscope of Presentations.” This is the first of a three-part series sponsored by BioMedica Diagnostics of […]
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