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Standard treatment for hemophilia patients involves replacement of the missing clotting factor either as prophylaxis or on-demand therapy. However, as many as one third of hemophilia A patients ...
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.
These inhibitors often increase over time and inhibit the action of coagulation in the body. Recombinant factor VIIa, which is an activated form of factor VII, bypasses factors VIII and IX and causes coagulation without the need for factors VIII and IX. It may be used in acquired hemophilia patients with higher inhibitor titers. [15]
Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that ...
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.
Notably, GalNAc-siRNAs are being employed in Alnylam's ALN-TTRsc and ALN-AT3 RNAi therapeutic programs for the treatment of transthyretin-mediated amyloidosis (ATTR) and hemophilia and rare ...
Factor VIII, an essential blood coagulation protein, [12] is used as a medication to treat and prevent bleeding in people with hemophilia A and other causes of low factor VIII. [13] [14] Certain preparations may also be used in those with von Willebrand's disease. [14] It is given by slow injection into a vein. [13]
Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]
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