Omaveloxolone for the Treatment of Friedreich’s Ataxia

Riley Kessler, Sonal Sharma, David R Lynch, Omaveloxolone for the Treatment of Friedreich’s Ataxia, Published Online: Aug 9th 2023 touchREVIEWS in Neurology. 2023;19(2):Online ahead of journal publication. 

 This review discusses the underlying cellular pathology and proposed mechanism of omaveloxolone in FRDA. The MOXIe study is presented in detail, including a discussion of the challenges faced in clinical trials in FRDA, and rare diseases more broadly. Finally, other therapies under investigation are reviewed briefly.

Omaveloxolone for the Treatment of Friedreich’s Ataxia