Evaluating the efficacy of vatiquinone in preclinical models of Leigh syndrome and GPX4 deficiency

Evaluating the efficacy of vatiquinone in preclinical models of Leigh syndrome and GPX4 deficiency

Abstract Background Genetic mitochondrial diseases are a major challenge in modern medicine. These impact ~ 1:4,000 individuals and there are currently no effective therapies. Leigh syndrome is the most common pediatric presentation of mitochondrial disease. In humans, patients are often treated wit...

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Main Authors: Ernst-Bernhard Kayser, Michael Mulholland, Elizaveta A. Olkhova, Yihan Chen, Holly Coulson, Owen Cairns, Vivian Truong, Katerina James, Brittany M. Johnson, Allison Hanaford, Simon C. Johnson
Format: Article
Language:English
Published: BMC 2025-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mitochondrial disease
Antioxidant
Vatiquinone
EPI-743
ROS
Leigh syndrome
Online Access:https://doi.org/10.1186/s13023-025-03582-x
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https://doi.org/10.1186/s13023-025-03582-x

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