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- W3118924958 abstract "Friedreich ataxia is a slowly progressive neurodegenerative disorder leading to ataxia, dyscoordination, dysarthria and in many individuals vision and hearing loss. It is associated with cardiomyopathy, the leading cause of death in Friedreich ataxia (FRDA), diabetes and scoliosis. There are no approved therapies, but elucidation of the pathophysiology of FRDA suggest that agents that increase the activity of the transcription factor Nrf2 may provide a mechanism for ameliorating disease progression or severity. In this work, we review the evidence for use of omaveloxolone in FRDA from recent clinical trials. Though not at present approved for any indication, the present data suggest that this agent acting though increases in Nrf2 activity may provide a novel therapy for FRDA." @default.
- W3118924958 created "2021-01-18" @default.
- W3118924958 creator A5009736756 @default.
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- W3118924958 date "2021-04-01" @default.
- W3118924958 modified "2023-09-27" @default.
- W3118924958 title "Omaveloxolone: potential new agent for Friedreich ataxia" @default.
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- W3118924958 doi "https://doi.org/10.2217/nmt-2020-0057" @default.
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