Article
Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update.
Orphanet journal of rare diseases - 18 Jun 2022
Dispenzieri Angela, Coelho Teresa, Conceição Isabel, Waddington-Cruz Márcia, Wixner Jonas, Kristen Arnt V, Rapezzi Claudio, Planté-Bordeneuve Violaine, Gonzalez-Moreno Juan, Maurer Mathew S, Grogan Martha, Chapman Doug, Amass Leslie
Abstract excerpt
BACKGROUND: Transthyretin amyloidosis (ATTR amyloidosis) is a rare, life-threatening disease caused by the accumulation of variant or wild-type (ATTRwt amyloidosis) transthyretin amyloid fibrils in the heart, peripheral nerves, and other tissues and organs. METHODS: Established in 2007, the Transthyretin Amyloidosis Outcomes Survey (THAOS) is the largest ongoing, global, longitudinal observational study of...
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