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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

Lookup NU author(s): Emerita Professor Katherine Bushby

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Abstract

Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600-6000 live male births. Although guidelines are available for various aspects of DMD, comprehensive clinical care recommendations do not exist. The US Centers for Disease Control and Prevention selected 84 clinicians to develop care recommendations using the RAND Corporation-University of California Los Angeles Appropriateness Method. The DMD Care Considerations Working Group evaluated assessments and interventions used in the management of diagnostics, gastroenterology and nutrition, rehabilitation, and neuromuscular, psychosocial, cardiovascular, respiratory, orthopaedic, and surgical aspects of DMD. These recommendations, presented in two parts, are intended for the wide range of practitioners who care for individuals with DMD. They provide a framework for recognising the multisystern primary manifestations and secondary complications of DMD and for providing coordinated multidisciplinary care. In part 1 of this Review, we describe the methods used to generate the recommendations, and the overall perspective on care, pharmacological treatment, and psycho,social management.

Publication metadata

Author(s): Bushby K; Finkel R; Birnkrant DJ; Case LE; Clemens PR; Cripe L; Kaul A; Kinnett K; McDonald C; Pandya S; Poysky J; Shapiro F; Tomezsko J; Constantin C

Publication type: Review

Publication status: Published

Journal: Lancet Neurology

Year: 2010

Volume: 9

Issue: 1

Pages: 77-93

ISSN (print): 1474-4422

ISSN (electronic): 1474-4465

URL: http://dx.doi.org/10.1016/S1474-4422(09)70271-6

DOI: 10.1016/S1474-4422(09)70271-6

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