title: CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy.
abstract: BACKGROUND
  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.
authors.affiliation: Children's National Medical Center, George Washington University, Washington, DC, USA.
authors.forename: Christopher F
authors.initials: CF
authors.lastname: Spurney
authors.forename: Carolina Tesi
authors.initials: CT
authors.lastname: Rocha
authors.forename: Erik
authors.initials: E
authors.lastname: Henricson
authors.forename: Julaine
authors.initials: J
authors.lastname: Florence
authors.forename: Jill
authors.initials: J
authors.lastname: Mayhew
authors.forename: Ksenija
authors.initials: K
authors.lastname: Gorni
authors.forename: Livia
authors.initials: L
authors.lastname: Pasquali
authors.forename: Alan
authors.initials: A
authors.lastname: Pestronk
authors.forename: Gerard R
authors.initials: GR
authors.lastname: Martin
authors.forename: Fengming
authors.initials: F
authors.lastname: Hu
authors.forename: Lei
authors.initials: L
authors.lastname: Nie
authors.forename: Anne M
authors.initials: AM
authors.lastname: Connolly
authors.forename: Diana M
authors.initials: DM
authors.lastname: Escolar
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D010865:Pilot Projects; D011446:Prospective Studies; D016896:Treatment Outcome; D014451:Ubiquinone / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D014815:Vitamins / Q000627:therapeutic use*
chemical_list: D000305:Adrenal Cortex Hormones; D014815:Vitamins; D014451:Ubiquinone; C024989:coenzyme Q10