Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103

Detalhes bibliográficos
Autor(a) principal: Enns,Gregory M.
Data de Publicação: 2017
Outros Autores: Cohen,Bruce H.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Journal of Inborn Errors of Metabolism and Screening
Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100804
Resumo: Abstract Mitochondrial dysfunction results in the production of an abnormally high amount of reactive oxygen and nitrogen species, which results in redox imbalance and glutathione deficiency. Therapeutics such as EPI-743 (?-tocotrienol quinone) and RP103 (cysteamine bitartrate) have the theoretical potential to improve redox imbalance by increasing intracellular glutathione and are currently under investigation in multiple clinical trials. This review provides an update on the use of these compounds in clinical trials related to primary and secondary mitochondrial disorders. These clinical trials have not only provided hope to affected patients and their families and caregivers, but also will serve as important stepping stones for further studies as our understanding of mitochondrial disease pathogenesis continues to improve.
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spelling Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103mitochondrial diseaseEPI-743RP103cysteamineglutathioneAbstract Mitochondrial dysfunction results in the production of an abnormally high amount of reactive oxygen and nitrogen species, which results in redox imbalance and glutathione deficiency. Therapeutics such as EPI-743 (?-tocotrienol quinone) and RP103 (cysteamine bitartrate) have the theoretical potential to improve redox imbalance by increasing intracellular glutathione and are currently under investigation in multiple clinical trials. This review provides an update on the use of these compounds in clinical trials related to primary and secondary mitochondrial disorders. These clinical trials have not only provided hope to affected patients and their families and caregivers, but also will serve as important stepping stones for further studies as our understanding of mitochondrial disease pathogenesis continues to improve.Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT)2017-01-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100804Journal of Inborn Errors of Metabolism and Screening v.5 2017reponame:Journal of Inborn Errors of Metabolism and Screeninginstname:Instituto Genética para Todos (IGPT)instacron:IGPT10.1177/2326409817733013info:eu-repo/semantics/openAccessEnns,Gregory M.Cohen,Bruce H.eng2019-05-14T00:00:00Zoai:scielo:S2326-45942017000100804Revistahttp://jiems-journal.org/ONGhttps://old.scielo.br/oai/scielo-oai.phpjiems@jiems-journal.org||rgiugliani@hcpa.edu.br2326-45942326-4594opendoar:2019-05-14T00:00Journal of Inborn Errors of Metabolism and Screening - Instituto Genética para Todos (IGPT)false
dc.title.none.fl_str_mv Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
title Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
spellingShingle Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
Enns,Gregory M.
mitochondrial disease
EPI-743
RP103
cysteamine
glutathione
title_short Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
title_full Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
title_fullStr Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
title_full_unstemmed Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
title_sort Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103
author Enns,Gregory M.
author_facet Enns,Gregory M.
Cohen,Bruce H.
author_role author
author2 Cohen,Bruce H.
author2_role author
dc.contributor.author.fl_str_mv Enns,Gregory M.
Cohen,Bruce H.
dc.subject.por.fl_str_mv mitochondrial disease
EPI-743
RP103
cysteamine
glutathione
topic mitochondrial disease
EPI-743
RP103
cysteamine
glutathione
description Abstract Mitochondrial dysfunction results in the production of an abnormally high amount of reactive oxygen and nitrogen species, which results in redox imbalance and glutathione deficiency. Therapeutics such as EPI-743 (?-tocotrienol quinone) and RP103 (cysteamine bitartrate) have the theoretical potential to improve redox imbalance by increasing intracellular glutathione and are currently under investigation in multiple clinical trials. This review provides an update on the use of these compounds in clinical trials related to primary and secondary mitochondrial disorders. These clinical trials have not only provided hope to affected patients and their families and caregivers, but also will serve as important stepping stones for further studies as our understanding of mitochondrial disease pathogenesis continues to improve.
publishDate 2017
dc.date.none.fl_str_mv 2017-01-01
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100804
url http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100804
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 10.1177/2326409817733013
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv text/html
dc.publisher.none.fl_str_mv Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT)
publisher.none.fl_str_mv Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT)
dc.source.none.fl_str_mv Journal of Inborn Errors of Metabolism and Screening v.5 2017
reponame:Journal of Inborn Errors of Metabolism and Screening
instname:Instituto Genética para Todos (IGPT)
instacron:IGPT
instname_str Instituto Genética para Todos (IGPT)
instacron_str IGPT
institution IGPT
reponame_str Journal of Inborn Errors of Metabolism and Screening
collection Journal of Inborn Errors of Metabolism and Screening
repository.name.fl_str_mv Journal of Inborn Errors of Metabolism and Screening - Instituto Genética para Todos (IGPT)
repository.mail.fl_str_mv jiems@jiems-journal.org||rgiugliani@hcpa.edu.br
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