Clinical trials in mitochondrial disorders, an update.
Antioxidants
Mitochondria
Mitochondrial augmentation
Mitochondrial biogenesis
Randomized clinical trials
Journal
Molecular genetics and metabolism
ISSN: 1096-7206
Titre abrégé: Mol Genet Metab
Pays: United States
ID NLM: 9805456
Informations de publication
Date de publication:
Historique:
received:
14
08
2020
accepted:
02
10
2020
pubmed:
2
11
2020
medline:
1
7
2021
entrez:
1
11
2020
Statut:
ppublish
Résumé
Mitochondrial disorders comprise a molecular and clinically diverse group of diseases that are associated with mitochondrial dysfunction leading to multi-organ disease. With recent advances in molecular technologies, the understanding of the pathomechanisms of a growing list of mitochondrial disorders has been greatly expanded. However, the therapeutic approaches for mitochondrial disorders have lagged behind with treatment options limited mainly to symptom specific therapies and supportive measures. There is an increasing number of clinical trials in mitochondrial disorders aiming for more specific and effective therapies. This review will cover different treatment modalities currently used in mitochondrial disorders, focusing on recent and ongoing clinical trials.
Identifiants
pubmed: 33129691
pii: S1096-7192(20)30202-X
doi: 10.1016/j.ymgme.2020.10.002
pmc: PMC7537630
pii:
doi:
Substances chimiques
Antioxidants
0
DNA, Mitochondrial
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1-13Subventions
Organisme : NINDS NIH HHS
ID : U54 NS078059
Pays : United States
Informations de copyright
Copyright © 2020 Elsevier Inc. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of Competing Interest GRANT AWARD #: NIH-5U54-NS078059-09.
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