Dutch biotechnology company Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK) are seeking 3-18 year-old participants with Duchenne muscular dystrophy (DMD) for a large-scale study to determine the usual progression of Duchenne muscular dystrophy and aid clinical trials. Prosensa is the developer of drisapersen and other experimental compounds to treat DMD, and GSK has been involved in drisapersen’s development. The goal of this observational study is to characterise DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the ‘six-minute walk test’. Prosensa has enrolled 100 participants out of its anticipated 250. This study will foster greater understanding of the progression of this debilitating disease and will help explore new endpoints that could be used to expedite drug development. Participants will be seen every six months for three years (a total of seven visits), but they can choose to stop participating at any time and can leave the natural history study and move into a drug study if one is available. (There is no medication being tested in the natural history study.) The natural history study will i) determine the muscle strength and function of children and adolescents with DMD and its evolution over time ii) assess the quality of life for DMD patients iii) and measure proteins found in the blood and urine that may be linked to disease progression. For additional study details and for contact information for sites in Argentina, Belgium, Brazil, France, Germany, Italy, the Netherlands, Sweden, Turkey and the USA see A Prospective Natural History Study of Progression of Subjects with Duchenne Muscular Dystrophy
