Not long after I started working at the Hemophilia Federation of America (HFA), the organization hosted a congressional reception to help raise awareness about bleeding disorders. The bleeding disorders community must maintain its visibility among government decision-makers who help address important issues, such as ensuring our access to the…
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Is it starting again? Is hemophilia about to take over my world? For more than seven years, my family has enjoyed life with hemophilia in the background. My youngest son, Caeleb, is 15 and has severe hemophilia A with an inhibitor. His early years were filled with complications that led…
GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will make the preventive treatment for hemophilia A more accessible to patients in China. Coagulation factor VIII, or FVIII,…
A single dose of the investigational gene therapy SB-525 (giroctocogene fitelparvovec) continues to prevent bleeds and the need for prophylaxis, or preventive treatment, for at least two years in men with severe hemophilia A. These are the latest results from the five men given the highest dose of the…
Sevenfact (eptacog beta) is a bypassing agent that’s approved to treat bleeding episodes in people with hemophilia A or hemophilia B who developed inhibitors, or neutralizing antibodies, against clotting factor VIII (FVIII) or factor IX (FIX).
Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing countries via the World Federation of Hemophilia (WFH) Humanitarian Aid Program after Roche and its subsidiaries Chugai and Genentech joined the program. The donation will provide access to Hemlibra for…
Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…
The first participant was dosed with the investigational therapy marstacimab in a Phase 3 clinical trial — still recruiting eligible patients across the globe — in adolescents and adults with severe hemophilia A or B. Pfizer‘s marstacimab (PF‐06741086) is a human antibody that blocks a…
Hemophilia is a lifelong condition, both for my husband, Jared, and me. He’s had hemophilia B since he was born, and until a cure is found, it’s here to stay. While I don’t have hemophilia, we both must live with it. I imagine hemophilia to be like a…
Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes a median of almost three years after being treated, according to full data from the Phase 3 AFFINE clinical trial. These new data add to top-line…