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Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2017-07-10 , DOI: 10.1056/nejmoa1616569 K. John Pasi , Savita Rangarajan , Pencho Georgiev , Tim Mant , Michael D. Creagh , Toshko Lissitchkov , David Bevan , Steve Austin , Charles R. Hay , Inga Hegemann , Rashid Kazmi , Pratima Chowdary , Liana Gercheva-Kyuchukova , Vasily Mamonov , Margarita Timofeeva , Chang-Heok Soh , Pushkal Garg , Akshay Vaishnaw , Akin Akinc , Benny Sørensen , Margaret V. Ragni
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2017-07-10 , DOI: 10.1056/nejmoa1616569 K. John Pasi , Savita Rangarajan , Pencho Georgiev , Tim Mant , Michael D. Creagh , Toshko Lissitchkov , David Bevan , Steve Austin , Charles R. Hay , Inga Hegemann , Rashid Kazmi , Pratima Chowdary , Liana Gercheva-Kyuchukova , Vasily Mamonov , Margarita Timofeeva , Chang-Heok Soh , Pushkal Garg , Akshay Vaishnaw , Akin Akinc , Benny Sørensen , Margaret V. Ragni
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Once-monthly subcutaneous administration of fitusiran resulted in dose-dependent lowering of the antithrombin level and increased thrombin generation in participants with hemophilia A or B who did not have inhibitory alloantibodies. (Funded by Alnylam Pharmaceuticals; ClinicalTrials.gov number, NCT02035605.)
中文翻译：
RNAi治疗靶向血友病A或B中的抗凝血酶
每月一次皮下注射氟尿嘧啶可导致剂量依赖性的抗凝血酶水平降低，并导致没有抑制性同种抗体的血友病A或B参与者的凝血酶生成量增加。（由Alnylam Pharmaceuticals资助； ClinicalTrials.gov编号，NCT02035605。）
更新日期：2017-08-31
中文翻译：
RNAi治疗靶向血友病A或B中的抗凝血酶
每月一次皮下注射氟尿嘧啶可导致剂量依赖性的抗凝血酶水平降低，并导致没有抑制性同种抗体的血友病A或B参与者的凝血酶生成量增加。（由Alnylam Pharmaceuticals资助； ClinicalTrials.gov编号，NCT02035605。）
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