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Article

Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy.

Journal of neurology - 1 Nov 2013

Coelho Teresa, Maia Luis F, da Silva Ana Martins, Cruz Márcia W, Planté-Bordeneuve Violaine, Suhr Ole B, Conceiçao Isabel, Schmidt Hartmut H-J, Trigo Pedro, Kelly Jeffery W, Labaudinière Richard, Chan Jason, Packman Jeff, Grogan Donna R

Abstract excerpt

Tafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerability, and efficacy of tafamidis 20 mg once daily in 86 patients who earlier received blinded treatment with tafamidis...

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Identifiers

PMID: 23974642
DOI: 10.1007/s00415-013-7051-7
PMCID: PMC3825212

Genes

TTR
FAP

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy.PMID 23974642Read ↗

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