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

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Authors

David R. Lynch · Children's Hospital of Philadelphia

Angie Goldsberry · Reata Pharmaceuticals (United States)

Christian Rummey · Swiss Data Science Center

Jennifer Farmer · Friedreich's Ataxia Research Alliance

Sylvia Boesch · Innsbruck Medical University

Martin B. Delatycki · Victorian Clinical Genetics Services

Paola Giunti

Abstract

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.

Topics & Keywords

Genetic Neurodegenerative Diseases DNA Repair Mechanisms Mitochondrial Function and Pathology

Publication Details

Published in: Annals of Clinical and Translational Neurology

Volume 11, Issue 1, pp. 4-16

DOI: 10.1002/acn3.51897

Field-Weighted Citation Impact: 6.55

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

Authors

Abstract

Topics & Keywords

Publication Details