This workshop will be unique in that its primary purpose is to facilitate coordination of translational and clinical aspects of Niemann-Pick disease, type C (NPC) on an international basis.
Thus, the goals of this meeting are twofold: first, to promote interactions among basic scientists involved in preclinical testing of potential therapeutic agents in mouse models of NPC and clinical scientists studying NPC and, second, to promote interactions and collaborations between clinical scientists studying NPC.