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The aim of this second unique forum is to bring together some 200 participants from around the world, including scientists and clinicians, start-up leaders, and families of patients groups, to inform and strengthen exchange and cooperation. The outcomes of the first conference on mucopolysaccharidosis (MPS) organized by the Sanfilippo Foundation Switzerland in Geneva in 2011 were encouraging and resulted in new collaborations between teams of researchers working on MPS programs, with some enterprises deciding to unite their research activities and cooperate. Now, four years on, the Foundation has decided to reassess the developments of research programs towards a treatment.