Clinically meaningful MR endpoints sensitive to preataxic SCA1 and SCA3.

Jayashree ChandrasekaranEmilien PetitYoung-Woo ParkSophie Tezenas du MontcelJames M JoersDinesh K DeelchandMichal PovažanGuita BananRomain ValabreguePhilipp EhsesJennifer FaberPierrick CoupéChiadi U OnyikePeter B BarkerJeremy D SchmahmannEva-Maria RataiSub H SubramonyThomas H MareciKhalaf O BusharaHenry PaulsonAlexandra DurrThomas KlockgetherTetsuo AshizawaChristophe LengletGülin Öznull null

Published in: Annals of neurology (2022)

Changes in cerebellar afferent and efferent pathways underlie the earliest symptoms of both SCAs. MR metrics collected with a harmonized advanced protocol in the multi-site trial setting allow detecting disease effects in individuals before ataxia onset with potential clinical trial utility for subject stratification. This article is protected by copyright. All rights reserved.

Keyphrases

clinical trial
phase ii
phase iii
study protocol
contrast enhanced
randomized controlled trial
magnetic resonance
open label
early onset
double blind
magnetic resonance imaging
sleep quality
climate change
physical activity