The cost of Hemgenix (etranacogene dezaparvovec), a one-time hemophilia B gene therapy, will be covered for patients living in Canada through the country’s public healthcare system. The letter of intent between CSL Behring Canada, Hemgenix’s developer, and the pan-Canadian Pharmaceutical Alliance (pCPA) excludes Quebec. The company said…
News
Researchers have developed new versions of clotting factor IX (FIX) that may be used to create personalized treatments for people with hemophilia B — specifically, new replacement therapies designed to help more effectively control bleeding. According to the team, the development of these new FIX versions may serve to create both short-…
Novo Nordisk has submitted an application to the U.S. Food and Drug Administration seeking approval to market Mim8 (denecimig), an injection therapy for preventing bleeds in people with hemophilia AÂ with or without inhibitors. If approved, Mim8 would be sold as a single-use, prefilled disposable pen for subcutaneous,…
Nearly all adults with acquired hemophilia A (AHA) safely achieved complete remission with either standard or low doses of rituximab when combined with corticosteroids and cyclophosphamide, a small study shows. Key features of disease remission, including an increase in clotting factor levels and a reduction in inhibitor levels,…
With encouragement from the World Federation of Hemophilia (WFH) and its partners, the World Health Organization (WHO) updated its essential medicines lists to better align with international clinical guidelines for managing hemophilia and other bleeding disorders. These lists — the Essential Medicines List (EML) and the…
Precision Biologic has announced an update of its Cryocheck Chromogenic Factor VIII assay, a test used to help diagnose and manage hemophilia A. The update, which is expected to be commercially available in the coming months, will ensure that the assay’s accuracy is not compromised by a new…
For physically active men with severe hemophilia A, switching from their standard preventive treatment to Hemlibra (emicizumab-kxwh) can help reduce bleeding, a recent study suggests. This change not only improved their quality of life but also reduced the burden of treatment, allowing them to maintain their active lives.
Infection and related complications pose a serious risk to people with acquired hemophilia A (AHA), accounting for more than half of the deaths in a registry study in Spain. About 15% of participants in the study experienced at least one infection requiring treatment or hospitalization, the study found. About…
Symptomatic spinal stenosis — the narrowing of spaces within the spinal canal, which can put pressure on the spinal cord and nerves — may be a complication in aging people with hemophilia, a study in Ireland reports. The risk of developing the condition increased with age, but it was…
Delaying the start of immunosuppressive treatment in adults diagnosed with acquired hemophilia A who are receiving Hemlibra (emicizumab-kxwh) to prevent bleeding may reduce the risk of infections and extend survival, a clinical study found. Patients in the Phase 2 study (NCT04188639 received Hemlibra for 12 weeks while immunosuppression…
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