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T. Klopstock

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Open access Aug 2026

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

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.

V. Carelli, Martina Romagnoli, F. Fortuna et al. · 0 citations

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