Female hemophilia AÂ patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…
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Note: This story was updated May 8, 2023, to remove hemophilia as one of the specific indications the collaboration will focus on, as these have not yet been publicly disclosed. AstraZeneca and Sernova are teaming up for a preclinical research project that aims to develop new cell therapies…
2seventy bio announced that it has achieved a key preclinical milestone under its joint program with Novo Nordisk to develop a gene-editing tool to treat hemophilia A. Details of the “proof of concept” research that met this milestone — reported to be worth $15 million to the…
Patient dosing has been completed in a registrational Phase 3 clinical trial in China testing BBM-H901, Belief BioMed‘s (BBM)’s investigational gene therapy for hemophilia B, the company announced. BBM-H901 is designed to restore the production of factor IX (FIX) — the blood-clotting protein that is faulty or missing…
Switching to Rebinyn (nonacog beta pegol) from other standard or extended half-life replacement therapies led to “robust” reductions in annual treatment costs among hemophilia B patients in Canada, a study based on real-world data reported. Its findings add to an earlier and real-world analysis of patients who…
The first Blood Advocacy Week, which promotes activities to showcase the importance of maintaining a safe and continuous blood supply and advocate for changes in policies that strengthen blood supply chains in the U.S., will take place April 24–28. Hemorrhage, or bleeding, is the leading cause of preventable death…
Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who are positive for factor IX inhibitors and need a routine prophylactic to prevent bleeds or reduce their frequency. This is the first approval for the daily under-the-skin injectable therapy, which…
With an emphasis on patient advocacy, supporters of World Hemophilia Day will participate in a variety of activities on April 17 to heighten awareness of the disease and other bleeding disorders that are thought to affect 1 in 10,000 people globally. The World Federation of Hemophilia (WFH) organizes the…
Higher replacement factor doses and the use of preventive treatments are just two ways prescription practices for hemophilia have changed over time, according to a survey of specialists over 22 years. The surveys also show the impact of Hemlibra’s (emicizumab-KXWH) approval and showcase the expectations for gene therapy,…
About one-third of moderate-to-severely affected people with hemophilia perceived declines in their daily functional abilities over a period of three to five years, a Dutch study found. While no strong risk factors for such declines were identified, patients more likely to be affected were those who were obese, had…
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