Biologyarticle2026-08-27

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

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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.

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Authors: Valério Carelli, Martina Romagnoli, Filippo Fortuna, Claudio Fiorini, Leonardo Caporali, Thomas Klopstock, Chiara La Morgia, Anna Ghelli

Institutions: Ludwig-Maximilians-Universität München, University of Bologna, LMU Klinikum, German Center for Neurodegenerative Diseases, Azienda Socio Sanitaria Territoriale Lariana, Istituto delle Scienze Neurologiche di Bologna, GNA University, Friedrich Baur Stiftung