医学
抗凝血酶
不利影响
药代动力学
养生
药效学
安慰剂
内科学
凝血因子
皮下注射
剂量
药理学
肝素
病理
替代医学
作者
John Pasi,Savita Rangarajan,Pencho Georgiev,Tim Mant,Michael Desmond Creagh,Toshko Lissitchkov,David Bevan,Steve Austin,C. R. M. Hay,Inga Hegemann,Rashid Kazmi,Pratima Chowdary,Liana Gercheva,Vasily Mamonov,M.A. Timofeeva,Chang-Heok Soh,Pushkal Garg,Akshay Vaishnaw,Akin Akinc,Benny Sørensen,Margaret V. Ragni
标识
DOI:10.1056/nejmoa1616569
摘要
Current hemophilia treatment involves frequent intravenous infusions of clotting factors, which is associated with variable hemostatic protection, a high treatment burden, and a risk of the development of inhibitory alloantibodies. Fitusiran, an investigational RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is in development to address these and other limitations.
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