Shaila D. Ghanekar, W. W. Miller, C. Meyer, Kevin J Fenelon, Alvin Lacdao, T. Zesiewicz
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Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone
Abstract Friedreich’s Ataxia (FRDA) is a devastating and progressive ataxia, marked by mitochondrial dysfunction and oxidative stress. Nrf2 activators such as omaveloxolone (Omav) modulate antioxidative mechanisms, and thus may be viable therapeutic agents in FRDA.