转甲状腺素
RNA干扰
淀粉样变性
医学
癌症研究
核糖核酸
遗传学
生物
内科学
基因
作者
David Adams,Alejandra González‐Duarte,William O’Riordan,Chih‐Chao Yang,Mitsuharu Ueda,Arnt V. Kristen,Ivailo Tournev,Hartmut Schmidt,Teresa Coelho,John L. Berk,Kon-Ping Lin,Giuseppe Vita,Shahram Attarian,Violaine Planté‐Bordeneuve,Michelle M. Mezei,Josep M. Campistol,Juan Buades,Thomas H. Brannagan,Byoung J. Kim,Jeeyoung Oh
标识
DOI:10.1056/nejmoa1716153
摘要
In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. (Funded by Alnylam Pharmaceuticals; APOLLO ClinicalTrials.gov number, NCT01960348 .).
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