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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 ...
Story at a glance Hemophilia B occurs when patients lack sufficient levels of the blood protein factor IX. Some current treatments for the condition involve repeated infusions of the protein. But ...
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]
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 ...
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]
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
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