Current and emerging treatment options in the management of Friedreich ataxia

Michelangelo Mancuso, Daniele Orsucci, Anna Choub, Gabriele SicilianoDepartment of Neuroscience, Neurological Clinic, University of Pisa, Pisa, ItalyAbstract: Friedreich ataxia (FRDA) is the most common autosomal recessive ataxia. Oxidative damage within the mitochondria seems to have a key role in...

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Autores principales:	Michelangelo Mancuso, Daniele Orsucci, Anna Choub, et al
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2010
Materias:	Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429
Acceso en línea:	https://doaj.org/article/8c796c9f2a9c45cf9f70a36ecdc7b1a8
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spelling	oai:doaj.org-article:8c796c9f2a9c45cf9f70a36ecdc7b1a82021-12-02T03:09:32ZCurrent and emerging treatment options in the management of Friedreich ataxia1176-63281178-2021https://doaj.org/article/8c796c9f2a9c45cf9f70a36ecdc7b1a82010-07-01T00:00:00Zhttp://www.dovepress.com/current-and-emerging-treatment-options-in-the-management-of-friedreich-a4953https://doaj.org/toc/1176-6328https://doaj.org/toc/1178-2021Michelangelo Mancuso, Daniele Orsucci, Anna Choub, Gabriele SicilianoDepartment of Neuroscience, Neurological Clinic, University of Pisa, Pisa, ItalyAbstract: Friedreich ataxia (FRDA) is the most common autosomal recessive ataxia. Oxidative damage within the mitochondria seems to have a key role in the disease phenotype. Therefore, FRDA treatment options have been mostly directed at antioxidant protection against mitochondrial damage. Available evidence seems to suggest that patients with FRDA should be treated with idebenone, because it is well tolerated and may reduce cardiac hypertrophy and, at higher doses, also improve neurological function, but large controlled clinical trials are still needed. Alternatively, gene-based strategies for the treatment of FRDA may involve the development of small-molecules increasing frataxin gene transcription. Animal and human studies are strongly needed to assess whether any of the potential new treatment strategies, such as iron-chelating therapies or treatment with erythropoietin or histone deacetylase inhibitors and other gene-based strategies, may translate into an effective therapy for this devastating disorder. In this review, we try to provide an answer to some questions related to current and emerging treatment options in the management of FRDA.Keywords: frataxin, idebenone, oxidative stress Michelangelo MancusoDaniele OrsucciAnna Choubet alDove Medical PressarticleNeurosciences. Biological psychiatry. NeuropsychiatryRC321-571Neurology. Diseases of the nervous systemRC346-429ENNeuropsychiatric Disease and Treatment, Vol 2010, Iss Issue 1, Pp 491-499 (2010)
institution	DOAJ
collection	DOAJ
language	EN
topic	Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429
spellingShingle	Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429 Michelangelo Mancuso Daniele Orsucci Anna Choub et al Current and emerging treatment options in the management of Friedreich ataxia
description	Michelangelo Mancuso, Daniele Orsucci, Anna Choub, Gabriele SicilianoDepartment of Neuroscience, Neurological Clinic, University of Pisa, Pisa, ItalyAbstract: Friedreich ataxia (FRDA) is the most common autosomal recessive ataxia. Oxidative damage within the mitochondria seems to have a key role in the disease phenotype. Therefore, FRDA treatment options have been mostly directed at antioxidant protection against mitochondrial damage. Available evidence seems to suggest that patients with FRDA should be treated with idebenone, because it is well tolerated and may reduce cardiac hypertrophy and, at higher doses, also improve neurological function, but large controlled clinical trials are still needed. Alternatively, gene-based strategies for the treatment of FRDA may involve the development of small-molecules increasing frataxin gene transcription. Animal and human studies are strongly needed to assess whether any of the potential new treatment strategies, such as iron-chelating therapies or treatment with erythropoietin or histone deacetylase inhibitors and other gene-based strategies, may translate into an effective therapy for this devastating disorder. In this review, we try to provide an answer to some questions related to current and emerging treatment options in the management of FRDA.Keywords: frataxin, idebenone, oxidative stress
format	article
author	Michelangelo Mancuso Daniele Orsucci Anna Choub et al
author_facet	Michelangelo Mancuso Daniele Orsucci Anna Choub et al
author_sort	Michelangelo Mancuso
title	Current and emerging treatment options in the management of Friedreich ataxia
title_short	Current and emerging treatment options in the management of Friedreich ataxia
title_full	Current and emerging treatment options in the management of Friedreich ataxia
title_fullStr	Current and emerging treatment options in the management of Friedreich ataxia
title_full_unstemmed	Current and emerging treatment options in the management of Friedreich ataxia
title_sort	current and emerging treatment options in the management of friedreich ataxia
publisher	Dove Medical Press
publishDate	2010
url	https://doaj.org/article/8c796c9f2a9c45cf9f70a36ecdc7b1a8
work_keys_str_mv	AT michelangelomancuso currentandemergingtreatmentoptionsinthemanagementoffriedreichataxia AT danieleorsucci currentandemergingtreatmentoptionsinthemanagementoffriedreichataxia AT annachoub currentandemergingtreatmentoptionsinthemanagementoffriedreichataxia AT etal currentandemergingtreatmentoptionsinthemanagementoffriedreichataxia
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Current and emerging treatment options in the management of Friedreich ataxia

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