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Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020

Mitochondrial diseases (MDs) are a group of genetic disorders that may manifest with vast clinical heterogeneity in childhood or adulthood. These diseases are characterized by dysfunctional mitochondria and oxidative phosphorylation deficiency. Patients are usually treated with supportive and sympto...

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Autores principales: Gruosso, Francesco, Montano, Vincenzo, Simoncini, Costanza, Siciliano, Gabriele, Mancuso, Michelangelo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7794679/
https://www.ncbi.nlm.nih.gov/pubmed/33383961
http://dx.doi.org/10.3390/jcm10010094
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author Gruosso, Francesco
Montano, Vincenzo
Simoncini, Costanza
Siciliano, Gabriele
Mancuso, Michelangelo
author_facet Gruosso, Francesco
Montano, Vincenzo
Simoncini, Costanza
Siciliano, Gabriele
Mancuso, Michelangelo
author_sort Gruosso, Francesco
collection PubMed
description Mitochondrial diseases (MDs) are a group of genetic disorders that may manifest with vast clinical heterogeneity in childhood or adulthood. These diseases are characterized by dysfunctional mitochondria and oxidative phosphorylation deficiency. Patients are usually treated with supportive and symptomatic therapies due to the absence of a specific disease-modifying therapy. Management of patients with MDs is based on different therapeutical strategies, particularly the early treatment of organ-specific complications and the avoidance of catabolic stressors or toxic medication. In this review, we discuss the therapeutic management of MDs, supported by a revision of the literature, and provide an overview of the drugs that should be either avoided or carefully used both for the specific treatment of MDs and for the management of comorbidities these subjects may manifest. We finally discuss the latest therapies approved for the management of MDs and some ongoing clinical trials.
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spelling pubmed-77946792021-01-10 Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020 Gruosso, Francesco Montano, Vincenzo Simoncini, Costanza Siciliano, Gabriele Mancuso, Michelangelo J Clin Med Review Mitochondrial diseases (MDs) are a group of genetic disorders that may manifest with vast clinical heterogeneity in childhood or adulthood. These diseases are characterized by dysfunctional mitochondria and oxidative phosphorylation deficiency. Patients are usually treated with supportive and symptomatic therapies due to the absence of a specific disease-modifying therapy. Management of patients with MDs is based on different therapeutical strategies, particularly the early treatment of organ-specific complications and the avoidance of catabolic stressors or toxic medication. In this review, we discuss the therapeutic management of MDs, supported by a revision of the literature, and provide an overview of the drugs that should be either avoided or carefully used both for the specific treatment of MDs and for the management of comorbidities these subjects may manifest. We finally discuss the latest therapies approved for the management of MDs and some ongoing clinical trials. MDPI 2020-12-29 /pmc/articles/PMC7794679/ /pubmed/33383961 http://dx.doi.org/10.3390/jcm10010094 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 Review
Gruosso, Francesco
Montano, Vincenzo
Simoncini, Costanza
Siciliano, Gabriele
Mancuso, Michelangelo
Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title_full Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title_fullStr Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title_full_unstemmed Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title_short Therapeutical Management and Drug Safety in Mitochondrial Diseases—Update 2020
title_sort therapeutical management and drug safety in mitochondrial diseases—update 2020
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7794679/
https://www.ncbi.nlm.nih.gov/pubmed/33383961
http://dx.doi.org/10.3390/jcm10010094
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