[Duchenne muscular dystrophy: current aspects and perspectives on treatment].

INTRODUCTION AND DEVELOPMENT

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease of genetic origin that affects male children. It is characterized by progressive muscle deterioration which results in the patient becoming wheelchair-dependent until death from cardio-respiratory complications. A few years ago, DMD patients' life quality and expectancy were poor and treatment options limited; valuable recommendations that significantly delay the progress of the disease and improve the patient's life quality have been brought about recently. Numerous therapeutic approaches are now in development in order to correct the DMD genetic defect at molecular level. In the mean time, a comprehensive system to maintain patients in their best possible physical condition is needed.

CONCLUSIONS

Accurate detection of complications enables caregivers to determine which patients are at higher risk and to provide treatment accordingly. Nevertheless, all of these efforts are dependent on early clinical and molecular diagnosis, careful record of clinical changes and long-term follow-up of DMD patients. Furthermore, the involvement of multidisciplinary groups and the patient's family is essential in said interventions.