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Description: Vatiquinone (formerly also known as EPI-743), a para-benzoquinone analog being developed for inherited mitochondrial diseases, is a novel, potent and orally bioavailable cellular oxidative stress protectant, which could be potentially used to treat mitochondrial diseases. The mechanism of action of EPI-743 involves augmenting the synthesis of glutathione, optimizing metabolic control, enhancing the expression of genetic elements critical for cellular management of oxidative stress, and acting at the mitochondria to regulate electron transport. Leigh syndrome is a mitochondrial disease caused by respiratory chain deficiency, and there are no proven effective therapies. EPI-743 is a potent cellular oxidative stress protectant and results of clinical trials for mitochondrial diseases are accumulating.
References: Brain Dev. 2018 Feb; 40(2):145-149.
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