Could NQO1 genotypes impact the results of clinical trials with idebenone?

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ID: 326683
2026
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Abstract
Carelli et al. argue that new insights into how NQO1 influences the response to idebenone warrant reevaluating the results of previous clinical trials, including those in Friedreich ataxia and Duchenne muscular dystrophy, using patient stratification by NQO1 genotype.
Reference Key
openalex_W7204442011 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Valério Carelli, Martina Romagnoli, Filippo Fortuna, Claudio Fiorini, Leonardo Caporali, Thomas Klopstock, Chiara La Morgia, Anna Ghelli
Journal Brain research
Year 2026
DOI
10.1093/brain/awag291
URL
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