Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A. “As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from…
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Doctors discovered a rare combination of blood clotting disorders — acquired hemophilia A alongside an inherited disorder called Factor XII deficiency — in a 70-year-old man set to undergo a leg amputation due to foot gangrene. “This case highlights the importance of considering acquired hemophilia A in unexplained bleeding…
Half of the children in Chile with severe hemophilia A and B exhibit joint damage despite receiving preventive treatment with clotting factors, a study reports. Damage to the cartilage is the main sign of joint impairment in children older than 8 years, while synovitis, or inflammation of the synovial…
Treatment with the extended half-life (EHL) therapies Eloctate (efmoroctocog alfa) and Alprolix (eftrenonacog alfa) was found to safely and effectively control bleeding during major orthopedic surgeries in people with hemophilia A or B, according to real-world data from two Italian centers. The therapies helped patients maintain…
Hemophilia has a substantial impact on sexual function and well-being for many men with the disease, but they rarely discuss it with their doctors, a study from the Netherlands found. “To address this gap, tools designed to provide and assess information about sexual functioning and well-being to normalize the…
Men in South America with severe hemophilia A or B are hit with both direct and indirect costs related to the genetic disease, which also affect quality of life, a new study reports. As much as 99% of the direct costs of hemophilia were related to factor…
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…
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,…
