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The safety and effectiveness of etranacogene dezaparvovec were evaluated by the US Food and Drug Administration (FDA) in two studies of 57 adult men 18 to 75 years of age with severe or moderately severe hemophilia B. [7] Effectiveness was established based on decreases in the men's annualized bleeding rate (ABR). [7]
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]
Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency (haemophilia A). [3] Haemophilia B was first recognized as a distinct disease entity in 1952. [4]
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 ...
In the US, fidanacogene elaparvovec is indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy; or have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes; and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var ...
B-cell cancers Tacatuzumab tetraxetan: AFP-Cide: mab: humanized: alpha-fetoprotein: cancer Tadocizumab [101] Fab: humanized: integrin α IIb β 3: percutaneous coronary intervention: Tafasitamab [47] Monjuvi: mab: humanized (from mouse) CD19: Y: relapsed or refractory diffuse large B-cell lymphoma Talacotuzumab [26] mab: humanized: CD123 ...
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