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Emerging Therapies for Transthyretin Cardiac Amyloidosis

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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. In… Click to show full abstract

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. 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: transthyretin amyloidosis; therapies transthyretin; amyloidosis; emerging therapies; transthyretin cardiac; cardiac amyloidosis

Journal Title: Current Treatment Options in Cardiovascular Medicine
Year Published: 2019

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