Natural History of Friedreich's Ataxia: Heterogeneity of Neurological Progression and Consequences for Clinical Trial Design.

Christian Rummey Louise A CorbenMartin DelatyckiGeorge WilmotSub H Subramony Manuela CortiKhalaf BusharaAntoine Duquette Christopher M Gomez J Chad Hoyle Richard H RoxburghLauren SeebergerGrace Yoon Katherine Mathews Theresa ZesiewiczSusan PerlmanDavid R Lynch

Published in: Neurology (2022)

- Understanding of the diversity within Friedreich's ataxia populations and their patterns of functional decline provides an essential foundation for future clinical trial design including patient selection and facilitates the interpretation of the clinical relevance of progression detected in Friedreich's ataxia.

Keyphrases

clinical trial
early onset
phase ii
open label
double blind
study protocol
single cell
case report
phase iii
randomized controlled trial
placebo controlled