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.
|Autori:||Spurney CF; Rocha CT; Henricson E; Florence J; Mayhew J; Gorni K; Pasquali L; Pestronk A; Martin GR; Hu F; Nie L; Connolly AM|
|Titolo:||CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy.|
|Anno del prodotto:||2011|
|Digital Object Identifier (DOI):||10.1002/mus.22047.|
|Appare nelle tipologie:||1.1 Articolo in rivista|