Duchenne muscular dystrophy: state of the art on new therapeutic approaches

Key words: Duchenne muscular dystrophy, Survival, Standard of care, Therapies

Duchenne muscular dystrophy is an X-linked disorder with an incidence of 1 in 5,000 male live births and is the most common muscular dystrophy in childhood. The disease is characterized by progression of muscle weakness and contractures leading to loss of ambulation before 13 years of age. The heart and respiratory muscle involvement are the main factors responsible for the limitation of life expectancy. In the last few years the optimization of the standards of care has produced a progressive increase in survival. These aspects are even more important at this time when new therapies are entering the clinical practice. The article reports an update on new therapy development.