Ixinity (trenonacog alfa) has been approved by the U.S. Food and Drug Administration (FDA) for use in children younger than 12 with hemophilia B. This label expansion by the FDA means that the factor IX (FIX) replacement therapy, sold by Medexus Pharmaceuticals, can now be used in…
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Deciding whether to tell your employer you have hemophilia can feel overwhelming, especially if symptoms affect your work. Learn about your legal protections, how to request reasonable accommodations, and practical steps to prepare for a confident, solution-focused conversation.
BAY 94-9027, developed by Bayer, is an investigational treatment for severe hemophilia A. It is a PEGylated molecule of recombinant factor VIII in which the B-domain, a region within the factor, is deleted. The therapy is produced using a baby hamster kidney cell line (BHK-21), and has…
The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…
When administered at a high dose, a gene therapy for hemophilia B known as FLT180a seems able to restore the activity of clotting factor IX (FIX) within a normal range in men with moderate-to-severe disease, early data from Phase 1/2 trial show. The findings were announced in an oral presentation,…
Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy developed by Pfizer that was formerly approved for hemophilia B, was found to be safe and effective in reducing bleeding episodes and the need for factor replacement therapy in a small group of men. That’s according to results from a long-term…
Heart disease is the No. 1 killer of women. Yet all of the studies I found on hemophilia and heart disease have studied only men. But essential lessons for women are in these studies, too. Today, National Wear Red Day, which symbolizes support for women’s heart health,…
During my husband Jared’s recent hospitalization, I heard one particular sentence more times than I can count: “We’ll wait for Doc’s approval.” Jared was admitted for a burn on his hand, an injury that, on its own, often follows a fairly standard treatment path. But because he lives with…
I have to admit, I was captivated by Hemophilia News Today‘s Facebook page during the final days of December, eagerly following its countdown of the year’s top 10 most-read columns. It was an incredible honor to see that my columns secured eight of those 10 spots. Each one…
Last in a series. Read parts one and two. In the final installment of my three-part interview with Chip Hailey, 75, we explore what his life looks like after gene therapy. Chip shares how Hemgenix (etranacogene dezaparvovec) has transformed his daily routine, his mindset, and his outlook…