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Aim: To investigate parent-reported health status of boys with Duchenne muscular dystrophy (DMD) compared with a large Australian normative population and a cohort of children with Charcot-Marie-Tooth disease type 1A (CMT1A). Methods: The Child Health Questionnaire parent form (CHQ-PF50) was completed by parents of 34 boys with confirmed DMD. Seventeen parents were followed up at 6 months. CHQ-PF50 data were compared with 2620 age-matched norms and 90 children with CMT1A. Results: All domains of the CHQ-PF50 for the DMD cohort were significantly lower than the general paediatric population, particularly for physical functioning ( t=−17.2, P < 0.001) and the child's ability to fulfil school and social roles because of physical limitations ( t=−9.4, P < 0.001). Parents experienced greatest emotional impact of their child's DMD around the time of loss of ambulation. Children with DMD had lower health status compared with children with CMT1A with the exception of the behaviour and pain domains. Physical functioning worsened during 6 months ( P= 0.04); no other changes in health status were observed at follow-up. Conclusions: Parents report the impact of DMD on health status to be considerably worse when compared with CMT1A. Interventions should target minimising the impact of physical limitations on role functioning. [ABSTRACT FROM AUTHOR]