Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy

Bibliographic Details
Main Author: Falcão De Campos, Catarina
Publication Date: 2023
Other Authors: Conceição, isabel
Format: Article
Language: eng
Source: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Download full: http://hdl.handle.net/10451/56479
Summary: © 2023 The Author(s). This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.
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spelling Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathyTTR stabilizerAmyloidosisFamilial amyloid polyneuropathyHereditary amyloid transthyretin amyloidosisTafamidisTransthyretin© 2023 The Author(s). This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.Hereditary amyloid transthyretin (ATTRv) amyloidosis is a devastating hereditary multisystemic disease affecting predominantly the peripheral and autonomic nervous systems and the heart. ATTRv is caused by mutations in the transthyretin (TTR) gene, leading to extracellular deposition of amyloid fibrils in multiple organs including the peripheral nervous system. If untreated, it is associated with a fatal outcome 10-12 years after disease onset. Different treatments are available for patients with ATTRv polyneuropathy. Tafamidis 20 mg is approved in Europe since 2011 for early stages of ATTRv polyneuropathy (stage I - able to walk without support) and it is recommended as first-line therapy in these patients. Tafamidis is a TTR stabilizer that selectively binds to TTR and kinetically stabilizes both wild-type native TTR and mutant TTR. Consequently, it has the potential to prevent the amyloidogenic cascade initiated by TTR tetramer dissociation into its monomers and subsequent misfolding and aggregation. Tafamidis is an oral drug, taken once per day, with proved efficacy, safety and tolerability in ATTRv-PN patients as demonstrated in different clinical trials and open-label extension studies as well in clinical practice setting with around 10 years of experience. Tafamidis treatment started in the earliest stages of the disease is associated with better neurological outcomes. A multidisciplinary approach in referral centres is also fundamental for monitoring patients to assess individual response to treatment.Dove PressRepositório da Universidade de LisboaFalcão De Campos, CatarinaConceição, isabel2023-02-28T12:10:18Z20232023-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10451/56479engDrug Healthc Patient Saf. 2023 Feb 17;15:51-6210.2147/DHPS.S3385771179-1365info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-11-08T17:04:05Zoai:repositorio.ul.pt:10451/56479Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T22:07:00.563517Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
title Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
spellingShingle Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
Falcão De Campos, Catarina
TTR stabilizer
Amyloidosis
Familial amyloid polyneuropathy
Hereditary amyloid transthyretin amyloidosis
Tafamidis
Transthyretin
title_short Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
title_full Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
title_fullStr Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
title_full_unstemmed Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
title_sort Updated evaluation of the safety, efficacy and tolerability of Tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy
author Falcão De Campos, Catarina
author_facet Falcão De Campos, Catarina
Conceição, isabel
author_role author
author2 Conceição, isabel
author2_role author
dc.contributor.none.fl_str_mv Repositório da Universidade de Lisboa
dc.contributor.author.fl_str_mv Falcão De Campos, Catarina
Conceição, isabel
dc.subject.por.fl_str_mv TTR stabilizer
Amyloidosis
Familial amyloid polyneuropathy
Hereditary amyloid transthyretin amyloidosis
Tafamidis
Transthyretin
topic TTR stabilizer
Amyloidosis
Familial amyloid polyneuropathy
Hereditary amyloid transthyretin amyloidosis
Tafamidis
Transthyretin
description © 2023 The Author(s). This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.
publishDate 2023
dc.date.none.fl_str_mv 2023-02-28T12:10:18Z
2023
2023-01-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10451/56479
url http://hdl.handle.net/10451/56479
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Drug Healthc Patient Saf. 2023 Feb 17;15:51-62
10.2147/DHPS.S338577
1179-1365
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Dove Press
publisher.none.fl_str_mv Dove Press
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron_str RCAAP
institution RCAAP
reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
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