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The National Bleeding Disorders Foundation (NBDF) is a United States patient advocacy organization for the care and treatment of inheritable blood and bleeding disorders such as hemophilia and von Willebrand disease. Founded in 1948, NBDF, then known as the National Hemophilia Foundation, helps secure funding for treatment centers and develops ...
Free Genetic Testing Initiative to be Available Nationally at Hemophilia Treatment Centers - My Life, Our Future pilot phase successfully completed - - Initiative aims to offer genotyping to all ...
Acquired haemophilia A (AHA) is a rare but potentially life-threatening bleeding disorder characterized by autoantibodies directed against coagulation factor VIII.These autoantibodies constitute the most common spontaneous inhibitor to any coagulation factor and may induce spontaneous bleeding in patients with no previous history of a bleeding disorder.
Up to 20% of people develop antibodies to the clotting factors which makes treatment more difficult. [3] The medication desmopressin may be used in those with mild haemophilia A. [16] Gene therapy treatment was in clinical trials as of 2022, with some approaches and products having received conditional approval. [17]
Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.
If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.
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