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

Christopher F. Spurney, Tesi Rocha Carolina, Erik K 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

29 Scopus citations

Abstract

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
Volume44
Issue number2
DOIs
StatePublished - Aug 2011
Externally publishedYes

Keywords

  • 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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