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Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects

Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplan...

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Autores principales: Russo, Massimo, Gentile, Luca, Toscano, Antonio, Aguennouz, M’Hammed, Vita, Giuseppe, Mazzeo, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7763612/
https://www.ncbi.nlm.nih.gov/pubmed/33316911
http://dx.doi.org/10.3390/brainsci10120952
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author Russo, Massimo
Gentile, Luca
Toscano, Antonio
Aguennouz, M’Hammed
Vita, Giuseppe
Mazzeo, Anna
author_facet Russo, Massimo
Gentile, Luca
Toscano, Antonio
Aguennouz, M’Hammed
Vita, Giuseppe
Mazzeo, Anna
author_sort Russo, Massimo
collection PubMed
description Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplantation (LT) and combined heart–liver transplantation, introduced in the 1990s, have been the only therapies for almost two decades. In 2011, tafamidis meglumine became the first specific drug approved by regulatory agencies, since then the attention toward this disease has progressively increased and several drugs with different mechanisms of action are now available. This review describes the drugs already on the market, those that have shown interesting results although not yet approved, and those currently being tested.
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spelling pubmed-77636122020-12-27 Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects Russo, Massimo Gentile, Luca Toscano, Antonio Aguennouz, M’Hammed Vita, Giuseppe Mazzeo, Anna Brain Sci Opinion Hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy is a progressive disease that is transmitted as an autosomal dominant trait and characterized by multiple organ failure, including axonal sensory-motor neuropathy, cardiac involvement, and autonomic dysfunction. Liver transplantation (LT) and combined heart–liver transplantation, introduced in the 1990s, have been the only therapies for almost two decades. In 2011, tafamidis meglumine became the first specific drug approved by regulatory agencies, since then the attention toward this disease has progressively increased and several drugs with different mechanisms of action are now available. This review describes the drugs already on the market, those that have shown interesting results although not yet approved, and those currently being tested. MDPI 2020-12-09 /pmc/articles/PMC7763612/ /pubmed/33316911 http://dx.doi.org/10.3390/brainsci10120952 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Opinion
Russo, Massimo
Gentile, Luca
Toscano, Antonio
Aguennouz, M’Hammed
Vita, Giuseppe
Mazzeo, Anna
Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title_full Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title_fullStr Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title_full_unstemmed Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title_short Advances in Treatment of ATTRv Amyloidosis: State of the Art and Future Prospects
title_sort advances in treatment of attrv amyloidosis: state of the art and future prospects
topic Opinion
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7763612/
https://www.ncbi.nlm.nih.gov/pubmed/33316911
http://dx.doi.org/10.3390/brainsci10120952
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