Neurobehavioral Profiles in Young Steroid-Naive Boys With Duchenne Muscular Dystrophy

BACKGROUND AND OBJECTIVES: Duchenne muscular dystrophy (DMD) is the most common pediatric hereditary neuromuscular disorder. While interest in brain involvement in DMD is increasing, neurobehavioral data in steroid-naive children remain limited, often nonstandardized, and derived from small cohorts. The primary objective was to characterize the baseline neurobehavioral profile of young steroid-naive boys with DMD enrolled in a multinational clinical trial. Secondary objectives were to examine associations with developmental features (speech and learning difficulties), motor function, parent respondent type, brain dystrophin isoform, age, and country. METHODS: We performed a baseline cross-sectional neurobehavioral analysis of steroid-naive boys recruited in the "Finding the Optimum Regimen for Duchenne Muscular Dystrophy" trial. Eligible participants were glucocorticoid-naive boys aged 4 to