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Article

Monitoring of asymptomatic family members at risk of hereditary transthyretin amyloidosis for early intervention with disease-modifying therapies.

Journal of the neurological sciences - 15 Jul 2020

Ueda Mitsuharu, Sekijima Yoshiki, Koike Haruki, Yamashita Taro, Yoshinaga Tsuneaki, Ishii Tomonori, Ando Yukio

Abstract excerpt

BACKGROUND: Hereditary transthyretin (ATTRv) amyloidosis is an adult-onset, systemic disorder caused by mutations in the transthyretin (TTR) gene. As ATTRv amyloidosis is inherited in an autosomal dominant manner, family members of the patients are at risk of developing the disease. METHODS: With an objective of discussing recommendations on monitoring of family members for early diagnosis of ATTRv amyloidosis,...

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PMID: 32353608
DOI: 10.1016/j.jns.2020.116813

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