Emerging Therapies for Transthyretin Cardiac Amyloidosis

被引：8

作者：

Alexander K.M. ^{[1
]}

Evangelisti A. ^{[1
]}

Witteles R.M. ^{[1
]}

机构：

[1] Stanford Amyloid Center, Division of Cardiovascular Medicine, Stanford University School of Medicine, 300 Pasteur Drive, Lane #158, Stanford, 94305, CA

来源：

Current Treatment Options in Cardiovascular Medicine | 2019年 / 21卷 / 8期

关键词：

Amyloidosis; Cardiomyopathy; Polyneuropathy; therapy; Transthyretin;

D O I：

10.1007/s11936-019-0743-2

中图分类号：

学科分类号：

摘要：

Purpose of review: Transthyretin cardiac amyloidosis is an underdiagnosed, undertreated disease which is associated with significant morbidity and mortality. This review will discuss the recent advancements in novel therapies for transthyretin amyloidosis. Recent findings: In recent phase 3 clinical trials, transthyretin stabilizers (tafamidis) and transthyretin silencers (patisiran and inotersen) have proven to be effective therapies for various forms of transthyretin amyloidosis. Summary: Understanding the recent and upcoming clinical trials for transthyretin amyloidosis will be important for improving the management of this challenging disease. © 2019, Springer Science+Business Media, LLC, part of Springer Nature.

引用

共 44 条

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