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Article

Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial.

Neurology - 21 Aug 2012

Coelho Teresa, Maia Luis F, Martins da Silva Ana, Waddington Cruz Marcia, Planté-Bordeneuve Violaine, Lozeron Pierre, Suhr Ole B, Campistol Josep M, Conceição Isabel Maria, Schmidt Hartmut H-J, Trigo Pedro, Kelly Jeffery W, Labaudinière Richard, Chan Jason, Packman Jeff, Wilson Amy, Grogan Donna R

Abstract excerpt

OBJECTIVES: To evaluate the efficacy and safety of 18 months of tafamidis treatment in patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP). METHODS: In this randomized, double-blind trial, patients received tafamidis 20 mg QD or placebo. Coprimary endpoints were...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 22843282
DOI: 10.1212/WNL.0b013e3182661eb1
PMCID: PMC4098875

Genes

TTR
FAP

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-22843282-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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