Preventive treatment with factor VIII (FVIII) — the missing blood clotting protein in hemophilia A — was not fully effective at preventing MRI-detected joint changes in boys with severe disease, according to a Canadian study. Regular evaluation of joints by MRI or ultrasounds, however, may help in identifying those…
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Hemophilia is a genetic disorder, mainly affecting men, caused by a deficiency in clotting factors VIII (hemophilia A), factor IX (hemophilia B), or factor XI (hemophilia C). Although patients had been previously warned against physical exercises, there are in fact numerous benefits of physical exercise for hemophilia sufferers, according to hog.org. MORE: …
Hemophilia B patients living in urban mainland China have longer hospitalizations and significantly higher medical costs — including more expensive fees for coagulation factors concentrates — compared with patients with hemophilia A, a retrospective study shows. In fact, people with hemophilia B had inpatient medical costs more than…
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…
Note: This article was updated June 26, 2024, to clarify that the analysis of fitusiran in surgery was conducted in patients receiving prophylactic treatment with the therapy. The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults…
An experimental cell-based therapy using Sernova’s innovative medical device, Cell Pouch, safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and reduced bleeds in a mouse model of the disease. These are the findings of a study, “…
How to Explain Hemophilia to Children
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
The U.S. Food and Drug Administration (FDA) has approved Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors, Genentech announced. Hemlibra is first newly approved treatment for this patient group in almost 20 years, the…
The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can…
A major complication in scientists’ efforts to treat hemophilia A by replacing a missing or defective clotting factor is an immune response to the treatment. Researchers have discovered a connection between the body’s production of pro-inflammatory molecules known as cytokines and its immune response to clotting factor VIII therapy. They used…