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Updated: Jul 30, 2025

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
Published on: June 9, 2018
[Therapeutic Strategies for Hereditary Transthyretin Amyloidosis]
1Department of Neurology, Graduate School of Medical Sciences, Kumamoto University.
Abstract:
Disease-modifying therapies such as transthyretin (TTR) gene-silencing drugs and TTR tetramer stabilizers have been developed for hereditary transthyretin (ATTR) amyloidosis, a major type of autosomal dominant hereditary amyloidosis. Recently, a second-generation TTR gene-silencing drug, vutrisiran, was approved for treating patients with hereditary ATTR amyloidosis in Japan. This new drug significantly reduced the patient's physical burden.
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