Lynch, D.R., Chin, M.P., Delatycki, M.B., Subramony, S., Corti, M., Hoyle, J.C., Boesch, S., Nachbauer, W., Mariotti, C., Mathews, K.D., Giunti, P., Wilmot, G., Zesiewicz, T., Perlman, S., Goldsberry, A., O'Grady, M. and Meyer, C.J. (2020). Ann Neurol. Accepted Author Manuscript. doi:10.1002/ana.25934
In the MOXIe trial, omaveloxolone significantly improved neurological function compared to placebo and was generally safe and well tolerated. It represents a potential therapeutic agent in FRDA.