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Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone

Authors Ghanekar SD, Miller WW, Meyer CJ, Fenelon KJ, Lacdao A, Zesiewicz TA

Received 22 March 2019

Accepted for publication 6 September 2019

Published 15 October 2019 Volume 2019:9 Pages 103—107


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Prof. Dr. Thomas Müller

Shaila D Ghanekar,1,2 Wai Wai Miller,1–3 Colin J Meyer,4 Kevin J Fenelon,1,2 Alvin Lacdao,1,2 Theresa A Zesiewicz1–3,5 

1University of South Florida (USF), Department of Neurology, Tampa, FL, USA; 2USF Ataxia Research Center, Department of Neurology, Tampa, FL, USA; 3USF Movement Disorders Neuromodulation Center, Department of Neurology, Tampa, FL, USA; 4Reata Pharmaceuticals, Inc., Department of Product Development, Irving, TX, USA; 5James A. Haley Veterans’ Hospital, Department of Neurology, Tampa, FL, USA

Correspondence: Theresa A Zesiewicz
University of South Florida, Department of Neurology, USF Ataxia Research Center, Tampa, FL, USA
Tel +1 813 974 5909
Fax +1 813 974 8032

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.

Keywords: FRDA, omaveloxolone, Nrf2 activators, oxidative stress, Omav

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