Switching to extended half-life (EHL) products has provided a reduction in the number of infusions and fewer bleeding events compared to standard treatments, particularly in hemophilia B, according to early real-world data from Europe. The study, “Real‐life experience in switching to new extended half‐life products…
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The U.S. Food and Drug Administration (FDA) has cleared for use CRYOcheck Chromogenic Factor VIII, Precision BioLogic‘s lab test to monitor factor VIII activity in the blood of hemophilia A patients ages 2 and older and help in disease management, the company said in a…
Shire has asked the U.S. Food and Drug Administration to grant Investigational New Drug status to its hemophilia A treatment SHP654. The gene therapy helps generate a clotting protein known as factor VIII that is missing or defective in hemophilia A. Shire said an FDA approval of its request will…
Fc fusion is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia.
In this series of podcasts from Bloodstream Media and shared by Bloodfeed, host Patrick James Lynch submits questions about hemophilia and other bleeding disorders from listeners to various hemophilia and health care experts. MORE: The Hemophilia CHOICE Project Survey The podcasts feature a pediatric hematologist, Dr. Arash…
A team of U.S. scientists discovered a naturally occurring F8 gene mutation that leads to increased activity of the factor VIII (FVIII) coagulation factor and enhanced blood clotting, offering new insights into the treatment of hemophilia A. The mutation was identified in a young man susceptible to excessive blood…
Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…
Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports. The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing…
Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…