Patient-reported outcomes in Friedreich's ataxia after withdrawal from idebenone.

Arron CookSylvia BoeschSuzette HeckEwout BruntThomas KlockgetherLudger SchölsAngela SchulzPaola Giunti

Published in: Acta neurologica Scandinavica (2019)

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.

Keyphrases

patient reported outcomes
end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
prognostic factors
physical activity
depressive symptoms
big data
current status