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Reader response: Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy

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In an observational study conducted in a single country, Coelho et al.1 reported favorable survival of patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) who underwent liver transplantation (LTx) or received… Click to show full abstract

In an observational study conducted in a single country, Coelho et al.1 reported favorable survival of patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) who underwent liver transplantation (LTx) or received tafamidis. This report is important to reconfirm the benefits of early treatments for patients with TTR-FAP.

Keywords: natural history; response natural; history survival; reader response; survival; transthyretin familial

Journal Title: Neurology
Year Published: 2019

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