An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy

Subject Inclusion Criteria

• Age: 5 - 11 years old
• Ambulant
• Diagnosis of DMD confirmed by at least one the following:

• Positive X-linked family history for typical Duchenne muscular dystrophy in older male relatives (onset by age 5 yr., wheelchair-bound by age 12 yr.) OR
• Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical Duchenne dystrophy OR
• Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical Duchenne dystrophy.
• On Glucocorticosteroids: Children must be on a steady dose of prednisone or deflazacort, on any schedule (Daily, alternate days, 10 days on, 10 days off or twice a week), for the last 6 months before starting the clinical trial. Dose of steroid or schedule cannot be altered during the study.
• Evidence of muscle weakness by MRC score or clinical functional evaluation
• Ability to provide reproducible repeat QMT bicep score within 10% of first assessment score.
• Ability to swallow tablets

Subject Exclusion Criteria

• Failure to achieve one or more of the diagnostic inclusion criteria cited above.
• Symptomatic DMD carrier
• Previous (6 months or less) or current use of Coenzyme Q10 (for DMD or any other disease)
• Use of carnitine, other amino acids, creatine, glutamine, or any herbal medicines within the last 3 months.
• History of significant concomitant illness or significant impairment of renal or hepatic function.