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Efanesoctocog alfa is a recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment & control of bleeding episodes; and perioperative management of bleeding. [1] [4]
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.
Joint capsule. Haemophilia A's phenotype has a quite wide range of symptoms encompassing both internal and external bleeding episodes. Individuals with more severe haemophilia tend to experience more intense and frequent bleeding, whereas those with mild haemophilia typically exhibit milder symptoms unless subjected to surgical procedures or significant trauma.
The efficacy and safety of concizumab were evaluated in a multi-national, multi-center, open-label, phase III trial (NCT04083781) with 91 adult and 42 adolescent male participants with hemophilia A or B with inhibitors who have been prescribed, or are in need of, treatment with therapies that bypass the inhibitor effect. [11]
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.
In 1998, the NHF convened the first Women with Bleeding Disorders Task Force, to address the difficulties women had in getting proper treatment. [5] In 2008, former NHF Board Chair and health advocate Val Bias became the group's CEO. [6] In 2013, actress and comedian Alex Borstein became the NHF's spokesperson for genetic testing. [7]
Most individuals who have Hemophilia B and experience symptoms are men. [6] The prevalence of Hemophilia B in the population is about one in 40,000; Hemophilia B represents about 15% of patients with hemophilia. [6] Many women carriers of the disease have no symptoms. [6] However, an estimated 10-25% of women carriers have mild symptoms; in ...
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]
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