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Updated: Jan 22, 2026

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
Published on: June 9, 2018
Emerging Therapies for Transthyretin Cardiac Amyloidosis
Kevin M Alexander1, Alessandro Evangelisti1, Ronald M Witteles2
1Stanford Amyloid Center, Division of Cardiovascular Medicine, Stanford University School of Medicine, 300 Pasteur Drive, Lane #158, Stanford, CA, 94305, USA.
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.
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