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Review
. 2019 Jul 15;21(8):40.
doi: 10.1007/s11936-019-0743-2.

Emerging Therapies for Transthyretin Cardiac Amyloidosis

Kevin M Alexander  1 , Alessandro Evangelisti  1 , Ronald M Witteles  2
Affiliations
Review

Emerging Therapies for Transthyretin Cardiac Amyloidosis

Kevin M Alexander et al. Curr Treat Options Cardiovasc Med. .

Abstract

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. Understanding the recent and upcoming clinical trials for transthyretin amyloidosis will be important for improving the management of this challenging disease.

Keywords: Amyloidosis; Cardiomyopathy; Polyneuropathy, therapy; Transthyretin.

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