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Initial Experience in the Treatment of Inherited Mitochondrial Disease with EPI-743

Inherited mitochondrial respiratory chain disorders are progressive, life-threatening conditions for which there are limited supportive treatment options and no approved drugs. Because of this unmet medical need, as well as the implication of mitochondrial dysfunction as a contributor to more common age-related and neurodegenerative disorders, mitochondrial diseases represent an important therapeutic target. 

Initial Experience in the Treatment of Inherited Mitochondrial Disease with EPI-743

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