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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 ] The gene is expressed in the liver to produce ...
Earlier this year, FDA approved Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or ...
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.
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. [2][3] This results in people bleeding for a longer time after ...
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] It is used to treat and prevent bleeding in hemophilia B if ...
The Food and Drug Administration approved Roctavian for adult patients with severe cases of hemophilia A, the inherited blood-clotting disorder that can lead to bleeding after minor injuries or ...
In hemophilia A and von Willebrand disease, it should only be used for mild to moderate cases. [1] It may be given in the nose, by injection into a vein, by mouth, or under the tongue. [1] Common side effects include headaches, diarrhea, and low blood sodium. [1] The low blood sodium that results may cause seizures. [1]
An antihemorrhagic (British English: antihaemorrhagic) agent is a substance that promotes hemostasis (stops bleeding). [1] It may also be known as a hemostatic (also spelled haemostatic) agent. [2] Antihemorrhagic agents used in medicine have various mechanisms of action: Systemic drugs work by inhibiting fibrinolysis or promoting coagulation ...
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