CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy

Muscle Nerve. 2011 Aug;44(2):174-8. doi: 10.1002/mus.22047. Epub 2011 Jun 22.

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.

Publication types

  • Clinical Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adrenal Cortex Hormones / therapeutic use*
  • Child
  • Child, Preschool
  • Disease Progression
  • Drug Therapy, Combination
  • Female
  • Humans
  • Male
  • Muscle Strength / drug effects
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Pilot Projects
  • Prospective Studies
  • Treatment Outcome
  • Ubiquinone / administration & dosage
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / therapeutic use
  • Vitamins / therapeutic use*

Substances

  • Adrenal Cortex Hormones
  • Vitamins
  • Ubiquinone
  • coenzyme Q10