Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis.

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第一作者： Arnt V,Kristen

第一单位： Department of Cardiology, University of Heidelberg, Heidelberg, D-69120, Germany.;Cardiovascular Center Darmstadt, Darmstadt, 64287, Germany.

作者： Arnt V,Kristen ^[1] ; Senda,Ajroud-Driss ^[2] ; Isabel,Conceição ^[3] ; Peter,Gorevic ^[4] ; Theodoros,Kyriakides ^[5] ; Laura,Obici ^[6]

作者单位： Department of Cardiology, University of Heidelberg, Heidelberg, D-69120, Germany.;Cardiovascular Center Darmstadt, Darmstadt, 64287, Germany. ^[1] Department of Neurology, Northwestern University, Feinberg School of Medicine, Chicago, IL 60611, USA. ^[2] Centro Hospitalar Lisboa Norte, Hospital de Santa Maria, Universidade de Lisboa, Faculdade de Medicina, 1649-028, Portugal. ^[3] Department of Medicine, Mount Sinai Medical Center, New York, NY 10029, USA. ^[4] The Cyprus Institute of Neurology & Genetics, Nicosia, 1683, Cyprus. ^[5] Amyloidosis Research & Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, 27100, Italy. ^[6]

关键词 cardiomyopathy clinical trials disease-modifying therapy hATTR amyloidosis patisiran polyneuropathy

主题词淀粉样神经病, 家族性(Amyloid Neuropathies, Familial);临床试验(主题)(Clinical Trials as Topic);人类(Humans);RNA, 小分子干扰(RNA, Small Interfering)

DOI 10.2217/nmt-2018-0033

PMID 30480471

发布时间 2020-08-18

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准确一般字义偏差语意偏离译文作用小提交