In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia B, and this translated into fewer bleeds each year. Levels of factor IX within the normal range…
For some patients with moderate or severe hemophilia, switching from standard to extended half-life replacement therapy results in improvements in key outcome measures, a study in Canada found. Most of these self-reported gains in health-related quality of life, and physical, mental, and social functioning three months after the switch,…
Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…
Nearly one-fifth of patients with mild hemophilia admitted to treatment centers in the U.S. are female, according to a large study of nearly 30,000 people. Yet, women and girls make up a smaller proportion of patients with severe or moderate hemophilia, ranging from less than 0.5% to a little…
Women who are carriers for hemophilia — meaning they can pass the disease-causing mutation to their children — think a more individually tailored, information-rich healthcare approach could improve the experience of getting or being pregnant, a small study found. “Healthcare professionals could use these insights to adapt their consultations to…
SCT800, a replacement therapy containing a man-made form of clotting factor VIII (FVIII), appears to be safe and effective at treating and preventing bleeds in adolescents and adults with severe hemophilia A, a study has found. The study, “Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted…
Voncento — a highly concentrated mixture of Von Willebrand factor (VWF) and clotting factor VIII (FVIII) — was seen to safely and effectively treat or prevent bleeds in children, age 12 and younger, with severe hemophilia A, according to data from a Phase 3 trial. Findings were reported in the…
Preventive, or prophylactic, treatment with Hemlibra (emicizumab) safely and effectively managed bleeding in adults and children with severe hemophilia A, a real-world study has found. The study, “Emicizumab prophylaxis: Prospective longitudinal real‐world follow‐up and monitoring,” was published in the journal Haemophilia. Originally developed by Roche’s subsidiary…
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