Prophylactic (preventive) treatment for hemophilia A and B is generally more effective than on-demand treatment in reducing the frequency of bleeding, but the benefit is not as clear in patients who have developed inhibitors that limit how well replacement clotting factors work. That’s according to real-world data from…
A single-center U.S. study found relatively little genetic testing was done for girls with a 50% risk of being a hemophilia carrier — people with the altered gene who don’t have hemophilia themselves but can pass it to their children — over a four-year period in Washington state.
Children with severe hemophilia A with blood group O are not at increased risk of developing neutralizing antibodies against factor VIII (FVIII) replacement therapy, a study found. The study, “Blood Group O Does Not Increase the Risk of Inhibitors in Severe Haemophilia A: Data from the…
Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy developed by Pfizer that was formerly approved for hemophilia B, was found to be safe and effective in reducing bleeding episodes and the need for factor replacement therapy in a small group of men. That’s according to results from a long-term…
Sobi and Sanofi’s philanthropic organization, Foundation S, have announced an additional donation of up to 100 million international units (IUs) of clotting factor treatment to support the World Federation of Hemophilia (WFH) Humanitarian Aid Program. This is the second renewal of the 10-year partnership that will continue…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes in people with both hemophilia A and hemophilia B, ages 12 and older. The therapy, already approved in the U.S. and the European Union, is designed to…
The first hemophilia B patient in Austria has been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to its developer, CSL Behring. “We are proud and grateful to offer hemophilia B patients in Austria a treatment option that has the potential to transform their lives,” Beate Natmessnig,…
The National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending the use of Altuvoct (efanesoctocog alfa) in England and Wales as an option for people with severe hemophilia A, ages 2 and older. The National Health Service, the U.K.’s public healthcare system, is…
World Hemophilia Day is April 17, and this year it will focus on the challenges of women and girls living with hemophilia and other bleeding disorders. The World Federation for Hemophilia (WFH) said the theme of its campaign is “Access for all: women and girls bleed too.” “Today,…
The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized…
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