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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]
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 ...
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 ...
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 ...
Biogen Idec (BIIB) and Swedish Orphan Biovitrum said a potential hemophilia B treatment has met safety goals in an early-stage study. The study involved 14 previously treated patients, and the ...
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]
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