The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should…
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Hympavzi (marstacimab-hncq) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, without inhibitors. It is given as weekly subcutaneous, or under-the-skin, injections that can be self-administered.
One of the most pervasive and amusing misconceptions about hemophilia is that a person with the condition could bleed to death from a tiny paper cut. People often think that those with hemophilia must avoid everything that is sharp, which isn’t entirely accurate. Hemophilia doesn’t cause people to…
The U.S. Food and Drug Administration (FDA) has approved Pfizer’s marstacimab under the brand name Hympavzi for use as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or B without inhibitors. With…
My 18-year-old son, Caeleb, is an artist. When he starts talking about colors, a lengthy conversation will ensue. Now in college majoring in digital arts, he’s passionate about his field, and his love of color runs deep. Art has been a saving grace for Caeleb, who lives with severe…
Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX (FIX), the clotting protein that is missing or faulty in hemophilia B, leading to about three times fewer bleeding episodes compared with prophylaxis, or preventive treatment, with regular infusions…
Ever since turning 30, I’ve been on a fast track toward self-improvement, determined to get the most out of life. I want to experience things meaningfully with my husband and daughter, doing things that bring me joy and fulfillment. But lately I’ve been feeling a bit disconnected from myself. That’s…
My grandmother always said, “If you don’t have anything good to say, don’t say anything at all.” She lived by those words, and in all of my memories, I can’t recall her ever speaking ill of anyone. Last week I found myself with nothing good to say, which left my…
Immunotherapy is an effective treatment strategy to suppress the production of autoantibodies and induce remission in people with acquired hemophilia A, a small study in China suggests. In some cases, however, multiple cycles of immunosuppressive treatment may be required for patients to achieve a good treatment response.The results were published…
Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that's approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.