轉(zhuǎn)甲狀腺素蛋白淀粉樣變心肌?。ˋTTR-CM）是一種罕見的心肌病，其愈后極差，目前臨床上缺乏早期診斷和有效的治療。主要介紹了該病的定義、病因、發(fā)病機制、主要治療手段，并按作用機制分類重點分析了目前已上市的ATTR-CM治療藥物他法米迪、伊諾特生、帕替司蘭和正在開發(fā)的ATTR-CM治療藥物acoramidis、vutrisiran、ION-682884等品種的特點及最新臨床研究結(jié)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

天津市科技基金項目（19YFZCSY00620）