The management of Duchenne muscular dystrophy

Abstract 

Duchenne muscular dystrophy (DMD) is a severe genetic disease with loss of walking at a mean age of 9.5 years and death in late teens or twenties. Despite major advances in understanding of the underlying molecular genetics and pathogenesis, curative treatment is not available. Physical therapy for prevention of contractures and promotion of ambulation is the mainstay of treatment. The provision of appropriate orthoses may facilitate and prolong walking. Nutritional management is aimed at avoiding both under-nutrition and obesity. Glucocorticoids have some benefit in DMD, but in view of the potential side-effects, the indications and pros and cons of their use should be discussed in full detail with the family. Early detection and treatment of respiratory and cardiac complications reduces morbidity, improves quality of life and prolongs survival.

The comprehensive management of these patients requires a multidisciplinary team approach, with one physician co-ordinating the overall care. While waiting for a curative treatment to become available, all symptomatic therapies should be used to ensure optimal condition of the patient.

Keywords: Duchenne muscular dystrophy, management

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• f1 Correspondence to: AYM. Tel:+44 (0)208 383 3295; Fax:+44 (0)208 740 3281; E-mail: a.manzur@ic.ac.uk