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Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…
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Treatment with an antibody designed to specifically block the anti-blood clotting, or anticoagulant, activity of an enzyme called activated protein C (APC) significantly reduced bleeding and related mortality in hemophilia A and hemophilia B mouse models, a study shows. The antibody, called HAPC1573, was also found to improve…
European authorities have recommended conditional marketing authorization for Roctavian (valoctocogene roxaparvovec), BioMarin Pharmaceutical’s one-time gene therapy for adults with severe hemophilia A. This positive decision, from the Committee for Medicinal Products for Human Use (CHMP), makes Roctavian the first gene therapy for hemophilia A to be recommended for European…
An innovative gel containing two snake venom proteins known to help blood clotting, rapidly and effectively forms stable clots and reduces bleeding even in the presence of a blood-clotting disorder, as is the case in hemophilia, a preclinical study shows. The gel, which transitions to a liquid form below…
Endometrial ablation was a difficult choice for columnist Jennifer Lynne, but it paid off by giving her respite from bleeding disorders.
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