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Efanesoctocog alfa is a recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment & control of bleeding episodes; and perioperative management of bleeding.
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]
Prothrombin complex concentrate (PCC), also known as factor IX complex, sold under the brand name Kcentra among others, is a combination medication made up of blood clotting factors II, IX, and X [9] (3-factor PCC) or, when also containing factor VII as does Kcentra, 4-factor PCC. [10]
Hympavzi is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered via
Since 2019, there’s been a spike in the number of children younger than 4 who were taken to the emergency room for ingesting gummy multivitamins and over-the-counter sleep aids like melatonin.
The companies expect the gene therapy to serve as a one-time shot to cut the rate of annual bleeding for hemophilia B patients, who represent about 15% of all patients with hemophilia.
Haemophilia (British English), or hemophilia (American English) [6] (from Ancient Greek αἷμα (haîma) 'blood' and φιλία (philía) 'love of'), [7] is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.
Emicizumab, sold under the brand name Hemlibra, is a humanized bispecific monoclonal antibody for the treatment of haemophilia A, developed by Genentech and Chugai (both organizations are subsidiaries of Hoffmann-La Roche). [4] A Phase I clinical trial found that it was well tolerated by healthy subjects. [5]
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