Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil
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 Tera...
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Academia Brasileira de Neurologia (ABNEURO)
2025-01-01
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| Series: | Arquivos de Neuro-Psiquiatria |
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| Online Access: | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1793936 |
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| author | Luiz Felipe Pinto Marcus V. Pinto Paula Accioli Gabriela Amorim Renata Gervais de Santa Rosa Moises Dias Mariana Guedes Carlos P. Gomez Roberto C. Pedrosa Marcia Waddington-Cruz |
| author_facet | Luiz Felipe Pinto Marcus V. Pinto Paula Accioli Gabriela Amorim Renata Gervais de Santa Rosa Moises Dias Mariana Guedes Carlos P. Gomez Roberto C. Pedrosa Marcia Waddington-Cruz |
| author_sort | Luiz Felipe Pinto |
| collection | DOAJ |
| description |
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). |
| format | Article |
| id | doaj-art-1d2ba974800046bcaf117c7756703c1e |
| institution | Kabale University |
| issn | 0004-282X 1678-4227 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Academia Brasileira de Neurologia (ABNEURO) |
| record_format | Article |
| series | Arquivos de Neuro-Psiquiatria |
| spelling | doaj-art-1d2ba974800046bcaf117c7756703c1e2025-01-15T23:30:31ZengAcademia Brasileira de Neurologia (ABNEURO)Arquivos de Neuro-Psiquiatria0004-282X1678-42272025-01-01830100100610.1055/s-0044-1793936Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in BrazilLuiz Felipe Pinto0https://orcid.org/0009-0009-1207-1459Marcus V. Pinto1https://orcid.org/0000-0002-9890-6657Paula Accioli2https://orcid.org/0009-0004-3341-7790Gabriela Amorim3https://orcid.org/0009-0001-6804-7687Renata Gervais de Santa Rosa4https://orcid.org/0000-0001-7739-6742Moises Dias5https://orcid.org/0009-0004-9540-7838Mariana Guedes6https://orcid.org/0000-0003-2730-3864Carlos P. Gomez7https://orcid.org/0000-0003-4565-7404Roberto C. Pedrosa8https://orcid.org/0000-0002-3270-1595Marcia Waddington-Cruz9https://orcid.org/0000-0003-4853-2236Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil.Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, Rio de Janeiro RJ, Brazil. 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).http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1793936PolyneuropathiesPrealbuminAmyloid Neuropathies, FamilialAmyloidosis, Familial |
| spellingShingle | Luiz Felipe Pinto Marcus V. Pinto Paula Accioli Gabriela Amorim Renata Gervais de Santa Rosa Moises Dias Mariana Guedes Carlos P. Gomez Roberto C. Pedrosa Marcia Waddington-Cruz Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil Arquivos de Neuro-Psiquiatria Polyneuropathies Prealbumin Amyloid Neuropathies, Familial Amyloidosis, Familial |
| title | Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil |
| title_full | Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil |
| title_fullStr | Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil |
| title_full_unstemmed | Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil |
| title_short | Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil |
| title_sort | real world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in brazil |
| topic | Polyneuropathies Prealbumin Amyloid Neuropathies, Familial Amyloidosis, Familial |
| url | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1793936 |
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