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Article

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis.

Journal of cardiovascular translational research - 1 Dec 2013

Merlini Giampaolo, Planté-Bordeneuve Violaine, Judge Daniel P, Schmidt Hartmut, Obici Laura, Perlini Stefano, Packman Jeff, Tripp Tara, Grogan Donna R

Abstract excerpt

This phase II, open-label, single-treatment arm study evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis. Twenty-one patients with eight different non-Val30Met mutations received 20 mg QD of tafamidis meglumine for 12 months. The primary outcome, TTR stabilization at Week 6, was achieved in 18 (94.7%) of 19 patients with evaluable data....

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Source

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Identifiers

PMID: 24101373
DOI: 10.1007/s12265-013-9512-x
PMCID: PMC3838581

Genes

TTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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