Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil.

doi:10.1055/s-0044-1793936

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Article

Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil.

Arquivos de neuro-psiquiatria - 1 Jan 2025

Pinto Luiz Felipe, Pinto Marcus V, Accioli Paula, Amorim Gabriela, Rosa Renata Gervais de Santa, Dias Moises, Guedes Mariana, Gomez Carlos P, Pedrosa Roberto C, Waddington-Cruz Marcia

Abstract excerpt

BACKGROUND: Tafamidis is a kinetic stabilizer that binds to the transthyretin (TTR) gene, inhibiting its dissociation. It is the only disease-modifying treatment for hereditary TTR amyloidosis with peripheral neuropathy (ATTRv-PN) available in the National Therapeutic Form (Formulário Terapêutico Nacional, FTN, in Portuguese) of the Brazilian Unified Health System (Sistema Único de Saúde, SUS, in Portuguese)....

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Topics

Humans
Female
Amyloid Neuropathies, Familial
Male
Retrospective Studies
Middle Aged

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Brazil

Benzoxazoles

Peripheral Nervous System Diseases

Adult

Treatment Outcome

Aged

Mutation

Source

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Identifiers

PMID: 39814002
DOI: 10.1055/s-0044-1793936
PMCID: PMC11735064

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