Background: Duchenne muscular dystrophy is one of the most severe muscle diseases to affect children. In the last twenty years, treatments have been established that have significantly improved patients' quality of life and life expectancy. The purpose of this article is to outline the main features of the disease and its treatment, and to examine possible future treatment options.
Method: The article is based on a literature search in PubMed, current international guidelines and our own clinical experience.
Results: Close monitoring by an interdisciplinary rehabilitation team forms the basis of treatment. Treatment with glucocorticoids can slow disease progression and improve motor function in the short term. The treatment may cause side effects, which must be monitored and which may require intervention. A not insignificant proportion of patients have cognitive and neuropsychiatric problems that must be addressed. Active intervention in response to signs of respiratory or cardiac failure is important. More causal treatment of Duchenne muscular dystrophy is under testing and offers cautious hope for future patients.
Interpretation: With improved treatment and increased life expectancy come new challenges for patients with Duchenne muscular dystrophy and their families, as well as new demands on the support services. This patient group requires close and comprehensive follow-up, also in the transition from child to adult.