Lead Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborators

Age

3 - 6 years

Sex

MALE

Healthy Volunteers

No

•Genetically confirmed dystrophin mutation compatible with DMD phenotype. Specifically, 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' OR showing complete absence of dystrophin by muscle biopsy.
•Ages between 3 years and \< 7 years
•Steroid-naïve
•Signed informed consent

•Treatment with CoenzymeQ10, creatine, amino acid supplements within 3 months of study entry
•Treatment with cardiac medications: beta-blockers, digoxin, and carvedilol
•Existing medical condition or physical disability that would alter subject's motor development
•Existing medical condition that precludes the use of corticosteroids
•Inability to swallow sample tablet in bite of soft food\*
•Investigator assessment that participant or family will not be compliant with treatment or study procedures
•Been on investigational DMD medication for the past 6 months