A natural anticoagulant protein called tissue factor pathway inhibitor (TFPI) is critical for defining the bleeding severity profiles of people with hemophilia A and B, researchers in France report. This discovery, which was found not to hold for healthy individuals, adds support to TFPI as an important element…
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Preventive treatment with Altuviiio (efanesoctocog alfa) provided better bleed prevention than Hemlibra (emicizumab) in adults and adolescents with hemophilia A, according to an indirect comparison of Phase 3 clinical trial data. “While it should be borne in mind that this is an indirect comparison and, as such,…
A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…
PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. These longer-lasting therapies don’t need to be administered as often, reducing the treatment burden for patients.
Blood levels of two proteins — C-reactive protein (CRP) and vascular endothelial growth factor (VEGF) — involved in inflammation and blood vessel formation are significantly associated with, and may help predict, acute bleeding in the joints of people with severe hemophilia A, a study has found. The study,…
BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year. The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270…
More than 12,300 hemophilia cases in developing countries were treated with therapy donations from Bioverativ and its partner Sobi in 2016, Bioverativ said in a press release about its participation in World Hemophilia Day. The company, which is promoting genetic testing in women and girls who could be susceptible to bleeding episodes, worked with other…
I had a lot to learn about hemophilia when I attended my first retreat for women with bleeding disorders in 2017. Some of the attendees were comparing their levels of clotting factor VIII (FVIII), the protein that’s deficient in hemophilia A, when I was shocked to learn mine were the lowest…
Read about Esperoct, an extended half-life infusion treatment approved to prevent or stop bleeding in children and adults with hemophilia A.
Hemlibra (emicizumab-KXWH) is an antibody-based therapy used to prevent or reduce bleeds in hemophilia A patients, with or without factor VIII (FVIII) inhibitors. It is given as an under-the-skin, or subcutaneous, injection.