Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.
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Hemophilia, a disorder characterized by excessive bleeding, is caused by the lack of activity of certain clotting factors, which are proteins that are needed to form blood clots.
There are several types of hemophilia, a rare bleeding disorder wherein the blood fails to clot properly. Hemophilia A and B are the most common types, while hemophilia C is comparatively rare.
Although no cure for hemophilia is currently available, a number of therapies can be used to control bleeding and other symptoms. These treatments can substantially increase life expectancy by lowering the risk of life-threatening complications such as bleeding in the brain, especially in people with severe hemophilia.
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…
The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…
Preventive treatment with Hemlibra (emicizumab) safely and effectively reduced bleeding rates in children with hemophilia A who were treated at a center in Texas, according to a new report. “We demonstrate a successful experience with emicizumab prophylaxis and safe [surgical] approach with a focus on minimizing postoperative bleeding,”…
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…
PEGylation is one of the molecular technologies that has been used to create extended half-life replacement therapies for hemophilia. These longer-lasting therapies don’t need to be administered as often, reducing the treatment burden for patients.
Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery. Esperoct received similar approval…