Note: This story was updated March 28, 2024, to clarify the gene therapy delivers the mRNA of the Cas9 enzyme. Intellia Therapeutics is ending its collaborative agreement with Regeneron, which had the two companies working together in developing hemophilia B gene-editing therapies, according to a securities and exchange…
Almost three years of treatment with SerpinPC was associated with a nearly complete resolution of bleeds and a reduction in the number of affected joints among men with severe hemophilia A or B who participated in a Phase 1/2a clinical trial. The…
Adding an antibody targeting the anticoagulant protein S to factor IX (FIX) replacement therapy accelerated blood clotting in samples from children with hemophilia B, including those with severe disease, a study showed. Greater production of the blood clotting protein thrombin also was seen in these samples, while blocking…
Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group. “The approval of Hemgenix in Canada…
Impairments in joint health are associated with certain functional deficits in the lower limbs among people with mild hemophilia, a study reports. A combination of aging, pain, and joint damage had a significant influence on patients’ performance in tests of muscle power, while aging alone explained most variation in…
Preventive treatment with Eloctate (efmoroctocog alfa) for up to five years led to sustained reductions in all bleed types for children, adolescents, and adults with severe hemophilia A, according to data from two Phase 3 trials and their open-label extension study. New analyses of these pivotal trials also…
About a quarter (24.4%) of men with hemophilia A and B seen at a clinic in Turkey met the clinical threshold for depression, and nearly three-quarters (74.4%) had anxiety, according to a recent analysis. Having a high school degree was linked to the presence of depression, whereas having…
A small percentage of young people with hemophilia A who are on preventive treatment with Hemlibra (emicizumab) experience severe muscle bleeds that require prolonged factor replacement therapy. These are the findings of a real-world, multicenter study in the U.S. which also show that most of these severe muscle…
The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…
The Hemophilia Federation of America (HFA) is now offering a “clinical trial finder” to help people with hemophilia and other bleeding disorders more easily connect with the studies they are eligible for. The platform enables visitors to search across all clinical trials, regardless of sponsor, for key…
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