Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses the important role of advocacy groups in rare diseases. One of the unique aspects of rare disease research has been the growing role of patient advocacy groups and the collaborative partnership that exists among such groups and the scientists who study rare diseases. This collaboration, which in many respects developed out of necessity on all sides, is unparalleled in other areas of medical research and product development. It has played a significant role in the adoption of public policies, available research funding, and the growing influence of the patient role in rare disease research.