Recent developments in the management of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is the most common paediatric muscle condition. Affected boys show first symptoms around the age of 2–5 years with progressive muscle weakness and wasting leading to severe disability and reduced life span due to cardiac and respiratory complications.Although no curative treatment is currently available for this severe and fatal condition, over the last years, advances in general care, corticosteroids and worldwide dissemination of standards of care especially in respiratory and cardiac care have changed the natural course of DMD. Careful and timely management of the disease, its complications and psychosocial aspects is mandatory. Such interventions have been shown to improve quality of life and prolong survival, so that most of patients now reach adulthood. Moreover, recent development of new therapeutic approaches and clinical trials require standardized treatment and management of these patients to evaluate the safety and efficacy of new compounds. The multi-systemic features of DMD require multidisciplinary team working, where paediatricians play an essential role in the management of patients, from diagnosis to planned transition into adult medical services.