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

Shaila D Ghanekar, Wai Wai Miller, Colin J Meyer, Kevin J Fenelon, Alvin Lacdao, Theresa A Zesiewicz; Degenerative Neurological and Neuromuscular Disease 15 October 2019 Volume 2019:9 Pages 103—107 doi:10.2147/DNND.S180027

Investigations into the pathological mechanisms of FRDA have revealed the central role of frataxin deficiency and the subsequent lack of transcription factor Nrf2. Consequently, the Nrf2 activators discussed here as well as the several treatments in development focus on reversing the oxidative stress environment caused by frataxin deficiency. While the discussed orphan drugs show promise in the treatment of FRDA, further investigations must be conducted in order to ensure the efficacy and safety of such drugs, as well as to determine even more therapeutic options.




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