Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.
Division of Hematology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
Blood. 2024 Sep 12;144(11):1230-1235. doi: 10.1182/blood.2023021944.
Emicizumab improves the procoagulant activity of select loss-of-function factor IX (FIX) variants with likely dysfunctional assembly of the intrinsic Xase complex, resulting in hemophilia B (HB). FVIII mimetics may represent an alternative nonfactor therapy for select patients with HB.
依库珠单抗提高了具有内在 Xase 复合物可能功能失调组装的特定失活功能因子 IX (FIX) 变体的促凝活性，从而导致乙型血友病 (HB)。FVIII 模拟物可能代表乙型血友病患者的另一种非因子替代疗法。
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