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Lookup NU author(s): Emerita Professor Katherine Bushby, Dr John Bourke, Dr Robert Bullock, Dr Michelle Eagle, Mark Gibson, Janice Quinby
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Duchenne muscular dystrophy (DMD) is an X-linked disorder for which there is currently no curative treatment. The natural history is such that affected boys need to use a wheelchair at around 9 years, develop respiratory and cardiac complications and die at a mean age of 19 years. While treatments based on gene modification or replacement are eagerly awaited, advances in medical management of DMD have made a significant difference to the natural history of the condition such that most affected individuals can now be expected to live into adulthood. The key interventions relate to the use of corticosteroids to improve muscle strength and function, surgical management of scoliosis and surveillance for and timely management of respiratory and cardiac complications. The predictable nature of the complications of DMD lends itself to the implementation of a planned programme of surveillance and management, which makes a real difference to survival and quality of life. © 2005 Elsevier Ltd. All rights reserved.
Author(s): Bushby K, Bourke J, Bullock R, Eagle M, Gibson M, Quinby J
Publication type: Article
Publication status: Published
Journal: Current Paediatrics
Year: 2005
Volume: 15
Issue: 4
Pages: 292-300
ISSN (print): 0957-5839
ISSN (electronic): 1878-206X
Publisher: The Medicine Publishing Company
URL: http://dx.doi.org/10.1016/j.cupe.2005.04.001
DOI: 10.1016/j.cupe.2005.04.001
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