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.
Authors
- Claudio Fiorini (ORCID: https://orcid.org/0000-0003-2099-0745)
- Anna Ghelli (ORCID: https://orcid.org/0000-0003-0830-0000)
- Thomas Klopstock (ORCID: https://orcid.org/0000-0003-2805-4652)
- Leonardo Caporali (ORCID: https://orcid.org/0000-0002-0666-4380)
- Martina Romagnoli (ORCID: https://orcid.org/0000-0003-1995-558X)
- Valério Carelli (ORCID: https://orcid.org/0000-0003-4923-6404)
- Filippo Fortuna
- Chiara La Morgia
Institutions
- German Center for Neurodegenerative Diseases (DE)
- LMU Klinikum (DE)
- GNA University (IN)
- Istituto delle Scienze Neurologiche di Bologna (IT)
- Azienda Socio Sanitaria Territoriale Lariana (IT)
- Friedrich Baur Stiftung (DE)
- Ludwig-Maximilians-Universität München (DE)
- University of Bologna (IT)
Publication Details
- Journal
- Brain
- Published
- 2026-08-27
- DOI
- https://doi.org/10.1093/brain/awag291
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Friedreich's Ataxia Research Alliance
- E-Rare