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A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor VIII (FVIII) coagulation factor and enhanced blood clotting, offering new insights into the treatment of hemophilia A. The mutation was identified in a young man susceptible to excessive blood…

Preventive treatment with Hemlibra (emicizumab) is associated with better bleeding control and lower medication costs compared with immune tolerance induction, or ITI, among children with severe hemophilia A with inhibitors, a new study in China found. The researchers noted that use of the injection therapy Hemlibra also led…

People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a global…

After a decade of living alongside my husband, Jared, and his severe hemophilia B, I’ve learned that not all bleeding is as straightforward as it may seem. Bleeds vary widely, not just in size but in severity and impact. What might look minor can be excruciating, while larger bleeds,…

The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…

The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective.