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Marstacimab is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people aged twelve years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors.
Concizumab, sold under the brand name Alhemo, is a monoclonal antibody used for the treatment of hemophilia A and hemophilia B. [5] [8] It is an anti-tissue factor pathway inhibitor. [5] [8] The most common adverse reactions include injection site reactions and hives (urticaria). [11]
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.
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] Studies of gene therapy are in early human trials. [17] Haemophilia A affects about 1 in 5,000–10,000, while haemophilia B affects about 1 in 40,000 males at birth.
Roctavian is the first gene replacement therapy for the most common form of hemophilia, enabling patients a way to forego or reduce the need for lifetime treatment with factor proteins needed to ...
U.S. officials on Thursday approved drugmaker BioMarin's gene therapy for the most common form of hemophilia, a $2.9 million infused treatment that can significantly reduce dangerous bleeding ...
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