Πέμπτη 18 Οκτωβρίου 2018

Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS).

In this report, we present data as assessed by the DNHS to support the prevalence of neurodevelopmental needs in young boys with DMD. To our knowledge, this is the first prospectively collected data of neurodevelopmental needs from a large well-characterized, internationally representative cohort of young boys with DMD. We also stratified boys with DMD mutations into three sub-categories based on DMD mutations likely to affect the dystrophin dp140 isoform to characterize in greater detail the neurodevelopmental needs in this cohort. By contrast, both Felisari et al.16 and Bardoni et al.17 evaluated cognitive impairment based on assessment of intellectual capacity through intelligence quotient, while our data is based on parent- or primary care-giver report. Speech delay was the most commo...

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