Low-dose immune tolerance induction (ITI) — a type of treatment that can be used to eliminate inhibitors that limit the effectiveness of replacement therapies — achieved partial success in 80% of children with severe hemophilia A and high inhibitor levels, according to a recent study. The study, “…
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Treatment with Idelvion (albutrepenonacog alfa) was effective in a real-world setting at controlling bleeds for five people with hemophilia B at a center in Spain. That’s according to data reported in the study, “Benefits of rIX-FP prophylaxis in patients with Haemophilia B: real-world evidence from…
G Shellye Horowitz explains how switching from on-demand treatment to prophylaxis helped her stay active and prevent repeated bleeds.
Afstyla (lonoctocog alfa) is an injectable factor replacement therapy used to prevent and treat bleeding episodes, including those occurring during surgery, in people with hemophilia A.
Transplants of embryonic stem (ES) cells that secrete the human factor VIII (FVIII) may be a promising therapeutic approach for patients with hemophilia A, according to an animal study conducted by researchers at the Nara Medical University School of Medicine in Japan. The study, “Therapeutic approaches for treating hemophilia…
A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that characterizes the disease in a mouse study. This potential new method might be a viable alternative for many clotting disorders. The study, “Systemic delivery of Factor IX messenger RNA…
I recently spent an afternoon with a group of parents raising young adults with hemophilia. They were part of a newer generation, people who grew up with a little more information, a little more community support, and slightly more medical options than what my husband, Jared, had access…
Pfizer’s Hympavzi (marstacimab) has been approved in the European Union (EU) to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with severe hemophilia A or hemophilia B who weigh at least 35 kg (77 pounds) and haven’t developed neutralizing…
Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old. Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer,…
Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A. They also discovered that hemophilia A…