A first patient has been dosed in XTEND-Kids, a Phase 3 clinical trial evaluating the safety and efficacy of the investigational replacement therapy efanesoctocog alfa in boys with severe hemophilia A. Hemophilia A is caused by the lack of a functional blood clotting protein called factor VIII…
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A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1…
Treatment with Hemlibra (emicizumab) can help to effectively manage bleeding in individuals with acquired hemophilia A (AHA), according to a new study. While clinical trials will need to be done to fully assess Hemlibra as an AHA therapy, the researchers said this medication also has the potential to…
Kevin Leurquin of Green Bay, Wisconsin, shares how living with hemophilia A has shaped his journey of learning, acceptance, and gratitude. In this video, he discusses staying active, protecting his health while racing and running, and using adaptation and creativity to manage daily life — from workouts to long hours…
Pfizer said the U.S. Food and Drug Administration (FDA) will review the company’s request to expand approval of its preventive treatment Hympavzi (marstacimab) to cover all people with hemophilia A or B ages 6 and older. Hympavzi is FDA-approved to treat hemophilia A and B in patients ages…
Combining immune tolerance induction, used to prevent the development of anti-factor VIII inhibitors, with Hemlibra (emicizumab) is a feasible and safe way of treating children with severe hemophilia A, a study shows. Published in the journal Haemophilia, the report covers clinical outcomes in the first seven patients to be…
Weekly use of the experimental, long-acting therapy efanesoctocog alfa safely and effectively prevents bleeds in adolescents and adults with severe hemophilia A, according to top-line, one-year data from the Phase 3 XTEND-1 trial. The therapy — being jointly developed by Sanofi and Sobi — was also superior to approved preventive therapies…
The National Hemophilia Foundation (NHF), through its Nursing Working Group (NWG), has released new guidelines to help hemophilia A patients self-administer Hemlibra (emicizumab). In its announcement, the foundation also highlighted the issuance of new guidelines for nurses to ensure safe and effective administration of standard into-the-vein…
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Anticoagulant Protein TFPI Crucial to Bleeding Severity Profile of Hemophilia Patients, Study Says
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…
A small percentage of young people with hemophilia A who are on preventive treatment with Hemlibra (emicizumab) experience severe muscle bleeds that require prolonged factor replacement therapy. These are the findings of a real-world, multicenter study in the U.S. which also show that most of these severe muscle…