Open AccessNeurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS).
Author(s) -
Mathula Thangarajh,
Christopher F. Spurney,
Heather GordishDressman,
Paula R. Clemens,
Eric P. Hoffman,
Craig McDonald,
E. Henricson,
Cinrg Investigators
Publication year - 2018
Publication title -
plos currents
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.282
H-Index - 49
ISSN - 2157-3999
DOI - 10.1371/currents.md.4cdeb6970e54034db2bc3dfa54b4d987
Subject(s) - duchenne muscular dystrophy , neurodevelopmental disorder , medicine , autism spectrum disorder , quality of life (healthcare) , intellectual disability , autism , population , muscular dystrophy , cognition , pediatrics , physical medicine and rehabilitation , physical therapy , psychology , psychiatry , nursing , environmental health
Duchenne muscular dystrophy (DMD) is the most common X-linked neuromuscular condition manifested by progressive skeletal muscle weakness, cardiopulmonary involvement and cognitive deficits. Neurodevelopmental symptoms and signs are under-appreciated in this population despite the recognition that cognition has a major impact on quality-of-life. We describe the neurodevelopmental needs in a large cohort of young boys with DMD from the DMD Natural History Study (DNHS). We explore the association between neurodevelopmental needs and DMD mutation location, and with glucocorticoid use. Methods: We prospectively evaluated 204 participants between ages 4 to less than 9 years of age with DMD as part of a large, longitudinal, international DNHS. We obtained parent- or primary care-giver report of neurodevelopmental needs as part of their study visit. We assessed the relationship between parent/care-giver neurodevelopmental needs and DMD mutation location, and glucocorticoid use.