Natural history in ambulant and non ambulant boys and young adults with Duchenne muscular dystrophy

The project aims to collect longitudinal data in a large cohort of ambulant and non-ambulant boys and young adults with Duchenne muscular dystrophy. As part of the clinical routine in all participating centers, all individuals with DMD are regularly followed using functional scales and other tools. As part of this project, all the available data will be collected in a repository, together with other relevant clinical and genetic information to create a large dataset. The possibility of having a very large dataset will allow to establish the progression of the disease in different age groups in relation to several factors that may modify the progression of the disease. This information will be useful when designing new clinical trials or to better understand the potential long-term efficacy of new treatments.