Long-term prophylaxis, or preventive treatment with Adynovate continues to safely reduce bleeding events over multiple years in children and adults with severe hemophilia A without causing neutralizing antibodies, a Phase 3b trial found. The study, “Long‐term safety and efficacy results from the phase…
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The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…
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What Life With Hemophilia Means to These Patients
In this video from the Bleeding Disorders Community, hemophilia patients and their families talk about how the disease has affected their lives but more importantly, how they haven’t let the disease redefine who they are as people. MORE: Three tips for coping with a hemophilia…
Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…
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What Does Hemophilia Look Like?
What does an illness look like? Sometimes a bald head, pale skin, or a mobility device are outward signs that someone has an illness. People would never know my sons have hemophilia by looking at them today. My oldest son, Julian, is 24. He lives on his own,…
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Explaining the Causes of Hemophilia
Hemophilia is a disease where a person blood lacks a factor which enables it to clot. This can lead to severe bleeding which can be life-threatening if left untreated. According to the World Federation of Hemophilia, there are three types of hemophilia: inherited, sporadic, and acquired. MORE: Five…
A new study from Finland demonstrated that neutralizing antibodies (inhibitors) can develop in people with untreated hemophilia, and that early intensive prevention might prevent bleeding and inhibitor development. The research report, titled “Inhibitor development in previously untreated patients with severe haemophilia A: a nationwide multicentre study in Finland,” was published in…
Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…
Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…
The safety and efficacy of emicizumab in patients with severe hemophilia A (HA) were recently evaluated in a study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” published in The New England Journal Of Medicine. Hemophilia A is caused by a lack of blood clotting factor…