Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data.

David R LynchAngie GoldsberryChristian RummeyJennifer FarmerSylvia BoeschMartin B DelatyckiPaola GiuntiJ Chad HoyleCaterina MariottiKatherine D MathewsWolfgang NachbauerSusan PerlmanSub H SubramonyGeorge WilmotTheresa ZesiewiczLisa WeissfeldColin Meyer

Published in: Annals of clinical and translational neurology (2023)

These results suggest a meaningful slowing of Friedreich ataxia progression with omaveloxolone, and consequently detail how propensity-matched analysis may contribute to understanding of effects of therapeutic agents. This demonstrates the direct value of natural history studies in clinical trial evaluations.

Keyphrases

clinical trial
early onset
electronic health record
big data
open label
randomized controlled trial
study protocol
phase ii
double blind
phase iii
deep learning
clinical evaluation