Telethon Italy was founded in 1990 by a patients’ association, the Italian Union Against Muscular Dystrophy (UILDM), with the aim of funding research on muscular dystrophies. Two years later, Telethon extended its mission to all human genetic diseases, meeting the urgent need of a multitude of families and patients affected by genetic disorders that were left behind by public and private investments. Since then, it has invested over 230 million euros in research. Fifteen years after its foundation, Telethon has assessed its research activities to fight muscular dystrophies and the other genetic diseases, and prepares for new ambitious challenges. We have achieved exciting results, as demonstrated by our successful gene therapy clinical trial and by the outstanding quality of our scientific publications. These achievements are largely due to our decision to fund excellent research, on the pure basis of merit, and strengthen our purpose towards constant improvement. To this aim, the Telethon’s Board of Directors and I have taken the decision to ask a Scientific Advisory Board composed by excellent independent scientists to help us in outlining a research strategic plan for the next five years. I am glad to present the Telethon strategic plan for research for years 2006-2010, which is focused on the concepts of excellence, therapy, flexibility and alliances. These are the keywords that will guide us to transform excellent science into therapies available to patients.