New standards of care and new therapies in Duchenne muscular dystrophy

Key words: Duchenne muscular dystrophy, Cardiac and respiratory complications, Survival, Standard of care

Duchenne muscular dystrophy has an incidence of 1 in 3,500 male live births and is the most common muscular dystrophy in childhood. The “typical” course of the disease is characterized by progression of muscle weakness and contractures leading to loss of ambulation before 13 years. The involvement of heart and respiratory muscles are responsible for progressive cardiac and respiratory impairment. In the last few years the introduction of standards of care leading to a better management of cardiac and respiratory complications has produced a progressive increase in survival. These aspects are even more important at the time new therapies are entering the clinical arena. In this article an update on standards of care and on the management of these patients as well as some information on new therapies are reported.