Abstract
Prader-Willi syndrome (PWS), initially described by Prader, Willi, and Labhart in 1956, is characterized by obesity, hypotonia, hyperphagia, delayed motor skill acquisition, short stature, mental retardation, hypothalamic dysfunction, and hypogonadism. This article reviews current knowledge regarding causes of and potential treatments for impaired growth, body composition, and physical function observed in children with PWS. Growth failure due to PWS has become an approved indication for growth hormone (GH) therapy. However, treatment of these children has raised awareness of other potential benefits of GH therapy, which in this particular group of patients may exceed linear growth promotion in importance. These include improvements in body composition, which leads to improved physical strength and function and increased energy expenditure.
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Acknowledgements
The authors wish to thank the important collaboration of Drs. Susan Meyers and Barbara Whitman, as well as the invaluable help of our study coordinator, Heidi Luebke MS. This work has supported in part by NIH grant M01 RR03186-13S1 as well as funding from Pharmacia, Genentech Foundation for Growth and Development, and Pfizer.
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Carrel, A., Allen, D.B. (2013). Growth Hormone Therapy in Children with Prader-Willi Syndrome. In: Radovick, S., MacGillivray, M. (eds) Pediatric Endocrinology. Contemporary Endocrinology. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-60761-395-4_6
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DOI: https://doi.org/10.1007/978-1-60761-395-4_6
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