Novo Nordisk is inviting children and young adults with hemophilia A and B to take part in its observational study of how this disease affects brain development, thinking, and behavior. Results will help researchers better understand how advances in hemophilia treatment, like routine prophylaxis, impact the brain by establishing…
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Hemophilia is a blood disorder that’s usually genetically inherited from parents. People born with hemophilia have little or no clotting factor – a protein needed for normal blood clotting. Hemophilia can also be acquired, meaning patients don’t inherit the disease from their parents but instead develop it at some point during…
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Hemophilia CHOICE Project Survey
The Hemophilia Foundation of American (HFA) has teamed up with the Centers for Disease Control and Prevention (CDC) to create a survey for hemophilia and bleeding disorder patients who don’t currently receive care from a government-funded hemophilia treatment center (HTC). MORE:Â Why shared data is important for furthering hemophilia research…
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Penn Researchers Coax Plants to Produce Human Clotting Factor for Hemophilia, Other Foreign Proteins
A research team from the departments of biochemistry and pathology at the University of Pennsylvania’s School of Dental Medicine has found a successful technique using genetic engineering to coax lettuce and tobacco plants to produce foreign proteins in their leaves. The applications include producing the human clotting factor to make…
Hemophilia B is a rare genetic bleeding disorder that impairs the blood’s ability to clot properly.
Interim results of Phase 3 clinical studies investigating two major challenges in hemophilia — the development of inhibitors (antibodies to the drug used to treat bleeding episodes) and the need for repeated venous injection of blood clotting protein Factor VIII (FVIII) — were recently released. The results were presented by…
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…
Creating personalized hemophilia B care is an important part of hemophilia B management.Your healthcare team can set up a treatment plan based on your specific needs and the severity of your symptoms.
Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…