| 转甲状腺素蛋白淀粉样变心肌病治疗药物的研究进展 |
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| 引用本文： | 罗西,马鑫,王维亭,田红. 转甲状腺素蛋白淀粉样变心肌病治疗药物的研究进展[J]. 现代药物与临床, 2020, 35(7): 1489-1494 |
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| 作者姓名： | 罗西 马鑫 王维亭 田红 |
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| 作者单位： | 天津市胸科医院, 天津 300051;天津药物研究院, 天津 300301 |
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| 基金项目： | 天津市科技基金项目（19YFZCSY00620） |
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| 摘 要： | 转甲状腺素蛋白淀粉样变心肌病(ATTR-CM)是一种罕见的心肌病,其愈后极差,目前临床上缺乏早期诊断和有效的治疗。主要介绍了该病的定义、病因、发病机制、主要治疗手段,并按作用机制分类重点分析了目前已上市的ATTR-CM治疗药物他法米迪、伊诺特生、帕替司兰和正在开发的ATTR-CM治疗药物acoramidis、vutrisiran、ION-682884等品种的特点及最新临床研究结果。
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| 关 键 词： | 转甲状腺素蛋白 淀粉样变性 心肌病 作用机制 |
| 收稿时间： | 2020-05-16 |
Research progress on drugs in treatment of transthyretin amyloidosis cardiomyopathy |
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| LUO Xi,MA Xin,WANG Wei-ting,TIAN Hong. Research progress on drugs in treatment of transthyretin amyloidosis cardiomyopathy[J]. Drugs & Clinic, 2020, 35(7): 1489-1494 |
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| Authors: | LUO Xi MA Xin WANG Wei-ting TIAN Hong |
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| Affiliation: | Tianjin Chest Hospital, Tianjin 300051, China;Tianjin Institute of Pharmaceutical Research Co. Ltd., Tianjin 300301, China |
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| Abstract: | Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare cardiomyopathy with a very poor prognosis. Currently, there is a lack of early diagnosis and effective treatment in the clinic. The definition, etiology, pathogenesis and main treatment of the disease are mainly introduced in this article, and the analysis of currently listed ATTR-CM therapeutic drugs tafamidis, inotersen, and patisiran, and the ATTR-CM therapeutic drugs acoramidis, vutrisiran, ION-682884, and other varieties under development and the latest clinical research results are focused on in this paper. |
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| Keywords: | transthyretin amyloidosis cardiomyopathies mechanism of action |
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