Wednesday, March 20, 2019

Patient reported outcomes in Friedreich's Ataxia after withdrawal from Idebenone

Cook, A. , Boesch, S. , Heck, S. , Brunt, E. , Klockgether, T. , Schöls, L. , Schulz, A. and Giunti, P. (2019). Acta Neurol Scand. Accepted Author Manuscript. doi:10.1111/ane.13088

This study provides no data to suggest that FRDA patients could correctly determine their treatment assignment over a 2‐month period. We hope that this study design will help inform future trials so that patients’ experiences of symptoms are more reliably measured.