Therapeutic Strategies for Hereditary Transthyretin Amyloidosis].

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.

Citation

Mitsuharu Ueda. Therapeutic Strategies for Hereditary Transthyretin Amyloidosis]. Brain and nerve = Shinkei kenkyu no shinpo. 2023 May;75(5):539-541

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PMID: 37194527

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