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Neurobehavioral Profiles in Young Steroid-Naive Boys With Duchenne Muscular Dystrophy: A Baseline Data Analysis From the FOR-DMD Trial - PubMed

a day ago
  • #Steroid-Naive
  • #Duchenne Muscular Dystrophy
  • #Neurobehavioral Profiles
  • Duchenne muscular dystrophy (DMD) commonly involves neurobehavioral issues, with about 40% of young steroid-naive boys experiencing speech difficulties and 25% learning difficulties.
  • Neurobehavioral assessments using parent-reported scales showed strong correlations, but only 5%-10% of boys exceeded screening cutoffs, indicating most did not meet formal diagnosis thresholds.
  • Speech and learning difficulties were linked to worse scores across all neurobehavioral scales, with classroom learning being the most frequently reported issue among those with significant impact.
  • No significant associations were found with age, brain dystrophin isoforms, or country, but weak correlations were observed between motor function and some psychosocial scales.
  • Mothers reported inattentive-overactive behaviors more frequently than fathers, highlighting potential differences in parent reporting.
  • The findings emphasize the need for routine early psychosocial screening and targeted interventions in DMD, even without formal neurobehavioral diagnoses.