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Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests

Wilson disease and alpha1-antitrypsin deficiency are two rare genetic diseases that may impact predominantly the liver and/or the brain, and the liver and/or the lung, respectively. The early diagnosis of these diseases is important in order to initiate a specific treatment, when available, ideally...

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Autores principales: Guillaud, Olivier, Dumortier, Jérôme, Couchonnal-Bedoya, Eduardo, Ruiz, Mathias
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9857715/
https://www.ncbi.nlm.nih.gov/pubmed/36673066
http://dx.doi.org/10.3390/diagnostics13020256
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author Guillaud, Olivier
Dumortier, Jérôme
Couchonnal-Bedoya, Eduardo
Ruiz, Mathias
author_facet Guillaud, Olivier
Dumortier, Jérôme
Couchonnal-Bedoya, Eduardo
Ruiz, Mathias
author_sort Guillaud, Olivier
collection PubMed
description Wilson disease and alpha1-antitrypsin deficiency are two rare genetic diseases that may impact predominantly the liver and/or the brain, and the liver and/or the lung, respectively. The early diagnosis of these diseases is important in order to initiate a specific treatment, when available, ideally before irreversible organ damage, but also to initiate family screening. This review focuses on the non-invasive diagnostic tests available for clinicians in both diseases. These tests are crucial at diagnosis to reduce the potential diagnostic delay and assess organ involvement. They also play a pivotal role during follow-up to monitor disease progression and evaluate treatment efficacy of current or emerging therapies.
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spelling pubmed-98577152023-01-21 Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests Guillaud, Olivier Dumortier, Jérôme Couchonnal-Bedoya, Eduardo Ruiz, Mathias Diagnostics (Basel) Review Wilson disease and alpha1-antitrypsin deficiency are two rare genetic diseases that may impact predominantly the liver and/or the brain, and the liver and/or the lung, respectively. The early diagnosis of these diseases is important in order to initiate a specific treatment, when available, ideally before irreversible organ damage, but also to initiate family screening. This review focuses on the non-invasive diagnostic tests available for clinicians in both diseases. These tests are crucial at diagnosis to reduce the potential diagnostic delay and assess organ involvement. They also play a pivotal role during follow-up to monitor disease progression and evaluate treatment efficacy of current or emerging therapies. MDPI 2023-01-10 /pmc/articles/PMC9857715/ /pubmed/36673066 http://dx.doi.org/10.3390/diagnostics13020256 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/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 (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Guillaud, Olivier
Dumortier, Jérôme
Couchonnal-Bedoya, Eduardo
Ruiz, Mathias
Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title_full Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title_fullStr Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title_full_unstemmed Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title_short Wilson Disease and Alpha1-Antitrypsin Deficiency: A Review of Non-Invasive Diagnostic Tests
title_sort wilson disease and alpha1-antitrypsin deficiency: a review of non-invasive diagnostic tests
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9857715/
https://www.ncbi.nlm.nih.gov/pubmed/36673066
http://dx.doi.org/10.3390/diagnostics13020256
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