Long term natural history in Duchenne muscular dystrophy

The aim of this project is to understand long term progression of functional changes in a large group of ambulant boys affected by Duchenne muscular dystrophy (DMD). Our network, including all the tertiary care centers in Italy, has followed over 150 boys affected by DMD for over 3 years, using the same assessments (outcome measures) used in the ongoing clinical trials. Following our group of patients for another 3 years will allow us to establish the effect of anumber of variables, such as age or level of performance at baseline on the progression of the disorder and will also provide the opportunity to identify early predictors of loss of ambulation. Long term follow up data has become increasingly important at the time there is the need to compare long term data from the first available data from clinical trials such as those using exon skipping that only used a control placebo group for their first year.