CINRG pilot trial of coenzyme Q10 in steroid‐treated duchenne muscular dystrophy

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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 subject‐ and 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. Muscle Nerve, 2011
Funding Information
  • Muscular Dystrophy Association (CNMC), PharmaNord (CNMC), General Clinical Research Center (M01RR020359)
  • National Institute of Health (NIH) (CNMC), National Center for Research Resources (UL-RR024992)
  • NCRR (K-23 RR16281-01)