Responsiveness of the SARA and natural history in 884 recessive and early onset ataxia patients.

Andreas TraschützAstrid D Adarmes-GomezMathieu AnheimJonathan BaetsBernard BraisCynthia GagnonJanina Gburek-AugustatSarah DossHasmet A HanagasiChristoph KammPeter KlivenyiThomas KlockgetherThomas KlopstockMartina MinneropAlexander MünchauMathilde RenaudFilippo Maria SantorelliLudger SchölsAndreas ThiemeStefan VielhaberBart P van de WarrenburgGinevra ZanniRalf-Dieter Hilgersnull nullMatthis Synofzik

Published in: Annals of neurology (2023)

This study comprehensively characterizes COA properties and annualized changes of the SARA across and within a large number of ataxias. It suggests specific approaches for optimizing its responsiveness that might facilitate regulatory qualification and trial design. This article is protected by copyright. All rights reserved.

Keyphrases

early onset
end stage renal disease
late onset
chronic kidney disease
ejection fraction
newly diagnosed
peritoneal dialysis
clinical trial
transcription factor
patient reported outcomes
phase iii
phase ii
intellectual disability
patient reported