CINRG pilot trial of coenzyme Q10 in Steroid-Treated duchenne muscular dystrophy

Christopher F. Spurney, Tesi Rocha Carolina, Erik Henricson, Julaine Florence, Jill Mayhew, Ksenija Gorni, Livia Pasquali, Alan Pestronk, Gerard R. Martin, Fengming Hu, Lei Nie, Anne M. Connolly, Diana M. Escolar

Research output: Contribution to journalArticlepeer-review


Introduction: Corticosteroid treatment slows disease progression and is the standard of care for Duchenne muscular dystrophy (DMD). Coenzyme Q10 (CoQ10) is a potent antioxidant that may improve function in dystrophin-deficient muscle. Methods: We performed an open-label, 'add-on' pilot study of CoQ10 in thirteen 5-10-year-old DMD patients on steroids. The primary outcome measure was the total quantitative muscle testing (QMT) score. Results: Twelve of 16 children (mean age 8.03 ± 1.64 years) completed the trial. Target serum levels of CoQ10 (≥2.5 μg/ml) were shown to be subjectand administration-dependent. Nine of 12 subjects showed an increase in total QMT score. Overall, CoQ10 treatment resulted in an 8.5% increase in muscle strength (P = 0.03). Conclusions: Addition of CoQ10 to prednisone therapy in DMD patients resulted in an increase in muscle strength. These results warrant a larger, controlled trial of CoQ10 in DMD

Original languageEnglish (US)
Pages (from-to)174-178
Number of pages5
JournalMuscle and Nerve
Issue number2
StatePublished - Aug 2011


  • Clinical trial
  • CoQ10
  • Duchenne muscular dystrophy
  • Muscle strength testing
  • Steroids

ASJC Scopus subject areas

  • Physiology
  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)


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