Mitochondrial disorders in children: toward development of small‐molecule treatment strategies

Mitochondrial disorders in children: toward development of small‐molecule treatment strategies

Abstract This review presents our current understanding of the pathophysiology and potential treatment strategies with respect to mitochondrial disease in children. We focus on pathologies due to mutations in nuclear DNA‐encoded structural and assembly factors of the mitochondrial oxidative phosphor...

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Bibliographic Details
Main Authors: Werner JH Koopman, Julien Beyrath, Cheuk‐Wing Fung, Saskia Koene, Richard J Rodenburg, Peter HGM Willems, Jan AM Smeitink
Format: Article
Language:English
Published: Springer Nature 2016-03-01
Series:EMBO Molecular Medicine
Subjects:
children
clinical trial
drug development
mitochondria
outcome measures
Online Access:https://doi.org/10.15252/emmm.201506131
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https://doi.org/10.15252/emmm.201506131

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