title: Short stature and pubertal delay in Duchenne muscular dystrophy.
abstract: Children with Duchenne muscular dystrophy (DMD) are shorter than their healthy peers. The introduction of corticosteroid (CS) has beneficial effects on muscle function but slows growth further and is associated with pubertal delay. In contrast to CS usage in most children and adolescents, weaning glucocorticoid is not a key objective of management in DMD. As the outlook for these young people improves, one of the main challenges is to reduce or offset the detrimental effects of CS on growth and development. This is a review of the aetiology and prevalence of short stature and delayed puberty in DMD, a summary of the treatments available and suggestions for areas of further research.
authors.affiliation: Department of Paediatric Endocrinology, Royal Victoria Infirmary, Newcastle upon Tyne, UK The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle upon Tyne, UK.
authors.forename: Claire L
authors.identifier: http://orcid.org/0000-0002-6084-5174
authors.initials: CL
authors.lastname: Wood
authors.affiliation: The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle upon Tyne, UK.
authors.forename: Volker
authors.initials: V
authors.lastname: Straub
authors.affiliation: The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle upon Tyne, UK.
authors.forename: Michela
authors.initials: M
authors.lastname: Guglieri
authors.affiliation: The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle upon Tyne, UK.
authors.forename: Kate
authors.initials: K
authors.lastname: Bushby
authors.affiliation: Department of Paediatric Endocrinology, Royal Victoria Infirmary, Newcastle upon Tyne, UK Institute of Genetic Medicine, Newcastle upon Tyne, UK.
authors.forename: Tim
authors.initials: T
authors.lastname: Cheetham
mesh_terms: D000293:Adolescent; D002648:Child; D005938:Glucocorticoids / Q000009:adverse effects / Q000627:therapeutic use; D006130:Growth Disorders / Q000139:chemically induced / Q000188:drug therapy / Q000209:etiology*; D019382:Human Growth Hormone / Q000627:therapeutic use; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000627:therapeutic use; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000150:complications* / Q000188:drug therapy; D011628:Puberty, Delayed / Q000139:chemically induced / Q000188:drug therapy / Q000209:etiology*; D013739:Testosterone / Q000627:therapeutic use
chemical_list: D005938:Glucocorticoids; D019382:Human Growth Hormone; D013739:Testosterone; D007334:Insulin-Like Growth Factor I
keywords: Endocrinology; Growth; Neuromuscular