Hemophilia is a genetic disorder that prevents blood clotting. Most common in men, there are two main types of hemophilia: hemophilia A and B. Below is a curated list of recommended reads for family members, friends and carers of patients with hemophilia, with help from Good Reads and Alibris.
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The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…
A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…
Social Clips
Explaining Hemophilia A and B
https://www.youtube.com/watch?v=S_YMGmHqIW4 This video from Claire Blatt shares a lecture about the effects of two blood-clotting disorders: hemophilia A and hemophilia B. MORE: A study shows that NovoSeven counters bleeding in children with hemophilia Though the lecture is aimed at nurses, it’s helpful for anyone looking to learn about these…
The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A. Wilate, developed and marketed by Octapharma, is an injectable medication that contains human plasma-derived von Willebrand Factor (VWF)…
Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that patients with hemophilia are generally young and that the disease is more common in Northeastern and Midwestern states. The study, “Occurrence rates of haemophilia among males in…
CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…
News
High Adherence to Prophylaxis Improves Physical Activity of Patients With Hemophilia A, Study Says
High adherence to prophylactic treatment not only reduces the number of spontaneous bleeding episodes in patients with hemophilia A, but it also improves their physical activity, according to a study. The study, “Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and…
Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…
My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in 2018.