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通过反义抑制前激肽释放酶控制遗传性血管性水肿
Antisense Inhibition of Prekallikrein to Control Hereditary Angioedema


Danny M. Cohn ... 其他 • 2020.09.24

摘要


遗传性血管性水肿的典型表现是反复发生不可预测且可能危及生命的皮下和黏膜肿胀发作。IONIS-PKK-LRx是一种配体结合型反义寡核苷酸,其设计目标是在受体介导下向肝细胞递送。在一项同情用药的先导性研究中,两例缓激肽介导的重度血管性水肿患者首先接受了为期12~16周的非结合型原型药物IONIS-PKKRx每周皮下注射给药,之后接受了为期7~8个月的IONIS-PKK-LRx给药(每3~4周给药1次,每次80 mg)。治疗伴有血管性水肿发作率降低(由阿姆斯特丹大学医学中心[Amsterdam UMC]资助)。





作者信息

Danny M. Cohn, M.D., Ph.D., Nicholas J. Viney, B.Sc., Lauré M. Fijen, M.D., Eugene Schneider, M.D., Veronica J. Alexander, Ph.D., Shuting Xia, M.S., Gwendolyn E. Kaeser, Ph.D., Charvi Nanavati, Ph.D., Brenda F. Baker, Ph.D., Richard S. Geary, Ph.D., Marcel Levi, M.D., Ph.D., Joost C.M. Meijers, Ph.D., and Erik S.G. Stroes, M.D., Ph.D.
From the Departments of Vascular Medicine (D.M.C., L.M.F., E.S.G.S.) and Experimental Vascular Medicine (J.C.M.M.), Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, and the Department of Molecular and Cellular Hemostasis, Sanquin Research (J.C.M.M.), Amsterdam; Ionis Pharmaceuticals, Carlsbad, CA (N.J.V., E.S., V.J.A., S.X., G.E.K., C.N., B.F.B., R.S.G.); and the Department of Medicine, University College London Hospitals NHS Foundation Trust, and the Cardiometabolic Programme, National Institute for Health Research University College London Hospitals and University College London Biomedical Research Centre, London (M.L.). Address reprint requests to Dr. Stroes at Amsterdam UMC, Department of Vascular Medicine, Meibergdreef 9, 1105 AZ Amsterdam, the Netherlands or at e.s.stroes@amsterdamumc.nl.

 

参考文献

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