CINRG PILOT TRIAL OF COENZYME Q10 IN STEROID-TREATED DUCHENNE MUSCULAR DYSTROPHY

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 mu g/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 44: 174-178, 2011