6th Annual Sanford-Burnham Rare Disease Day Symposium Treating Disease with Sugars February 27 - 28, 2015 | 10905 Road to the Cure, San Diego CA 92121 Scientific Symposium Simple sugars can treat patients with rare diseases, but these supplements can also impact aging, immune function, cancer and development of diabetes. Clinical trials are underway, but the underlying basic science remains obscure. February 27, 2015 9:00 am - 5:00 pm Participate either in person or online. “Doctor-is-in” Session Our program covers all of these areas and our speakers will offer some explanations. Clinicians, patients, and their advocates are invited to join bench scientists, present data, explore mysteries, advance theories and offer hope. Join us. February 28, 2015 9:00 am - 1:00 pm Interact with researchers and clinicians in an intimate, collaborative environment. N-acetylglucosamine: a Potential Therapy for Multiple Sclerosis Keeping it Sweet: Treatment Options for GNE Myopathy Michael Demetriou, M.D., Ph.D. May Christine Malicdan, M.D., Ph.D. Neurology School of Medicine, University of California, Irvine National Center for Neurology and Psychiatry, NIH Hexosamine Pathway Activation Improves Protein Quality Control and Extends Lifespan Mannose: Healer and Destroyer Martin Denzel, Ph.D. Sanford-Burnham Medical Research Institute Max Planck Institute for Biology of Ageing The Double Edged Sword of Nutrient Regulation of Protein Function by a Sugar Gerald (Jerry) Hart, Ph.D. Hudson Freeze, Ph.D. Nothing to Sneeze at: the Emerging Role of Sugars in Allergic Syndromes Jonathan Lyons, M.D. Laboratory of Allergic Diseases, NIAID/NIH Johns Hopkins University School of Medicine Letting Go While Holding On Jennifer Yashari, M.D. Testing the Waters: Using Zebrafish as a Tool to Study Mechanisms Underlying a Glycosylation Disorder Neuromuscular Disease Foundation Jamie Chu, M.D. Safety and Efficacy Data from a Randomized, Controlled Phase 2 Study (Including Long-Term 2 Year Data) of Sialic Acid Extended Release in patients with GNE Myopathy Mount Sinai’s Recanati/Miller Transplantation Institute Sunil Agarwal, M.D. Fucose: Targeting Melanoma with Sugar Eric Lau, Ph.D. Sanford-Burnham Medical Research Institute Ultragenyx Pharmaceutical Advancing ManNAc as a Therapy for GNE Myopathy Nuria Carrillo-Carrasco, M.D. National Center for Advancing Translational Sciences, NIH Registration and symposium updates sanfordburnham.org/rarediseaseday