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。

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.

R972

天津市科技基金项目（19YFZCSY00620）