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Three hemophilia B patients said they were able to lead a relatively normal life, including being more active and participating in sports, about three years after receiving the investigational gene therapy AMT-060 in a clinical trial. Their experiences were reported in the study, “The Patient…

Our hemophilia community has been here before. A novel virus — previously, it was HIV — hit the world, and our community was uncertain about how it would affect us. Patients were told to turn to doctors for help and guidance, but the information each patient received varied…

Greater awareness of hemophilia and its symptoms is associated with better adherence to treatment by patients, and those with more severe disease are most likely to use a treatment as prescribed, a study based on a new questionnaire shows. Responses were seen to validate this questionnaire, which addresses…

Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…

Due to the global COVID-19 pandemic, World Hemophilia Day (WHD) will be  a little different this year to keep everyone safe. Still, there are multiple ways to participate in the April 17 event intended to increase awareness and understanding of hemophilia and other bleeding disorders. Begun in 1989 and…

After receiving guidance from both the U.S. Food and Drug Administration and the European Medicines Agency, Catalyst Biosciences is preparing to launch a pivotal Phase 3 clinical trial assessing marzeptacog alfa activated (MarzAA) for the treatment of bleeding episodes in people hemophilia A or B with…