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Review Article

A Comprehensive Review on Chemistry and Biology of Tafamidis in Transthyretin Amyloidosis

Author(s): Monali B. Patil, Piyush Ghode* and Prashant Joshi*

Volume 24, Issue 6, 2024

Published on: 11 October, 2023

Page: [571 - 587] Pages: 17

DOI: 10.2174/0113895575241556231003055323

Price: $65

Abstract

Transthyretin amyloid cardiomyopathy and Transthyretin amyloid peripheral neuropathy are progressive disease conditions caused by Transthyretin amyloidosis (ATTR) fibril infiltration in the tissue. Transthyretin (TTR) protein misfolding and amyloid fibril deposits are pathological biomarkers of ATTR-related disorders. There are various treatment strategies targeting different stages in pathophysiology. One such strategy is TTR tetramer stabilization. Recently, a new TTR tetramer stabilizer, tafamidis, has been introduced that reduces the protein misfolding and amyloidosis and, consequently, disease progression in ATTR cardiomyopathy and peripheral neuropathy. This review will provide a comprehensive overview of the literature on tafamidis discovery, development, synthetic methods, pharmacokinetics, analytical methods and clinical trials. Overall, 7 synthetic methods, 5 analytical methods and 23 clinical trials have been summarized from the literature.

Keywords: Transthyretin amyloid cardiomyopathy, transthyretin amyloid peripheral neuropathy, tafamidis, synthetic methods, analytical techniques, pharmacokinetics and clinical trials.

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Graphical Abstract

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