WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium has grown to an international research conference that attracts over 2000 participants from more than 50 countries around the globe.
Course Director: Chester B. Whitley, PhD, MD, Chair
Lysosomal Disease Network
Director, Advanced Therapies
Director, Gene Therapy Center
Director, PKU Clinic Departments of Pediatrics,
and Experimental and Clinical Pharmacology,
University of Minnesota
516 Delaware St SE
Minneapolis, MN 55455
**Special thanks are given to members of the 2021 Program Committee responsible for abstract review and scoring for the 2021 platform presentations.
Brian Bigger, PhD**
Elizabeth Braunlin, MD, PhD**
Philip J. Brooks, PhD**
Amber R. Brown, CPP
Brenda M. Diethelm-Okita, MPA
Thomas Eggermann, MD, PhD**
Roberto Giugliani, MD, PhD, MSc**
Jeanine R. Jarnes, PharmD
Virginia Kimonis, MD**
R. Scott McIvor, PhD**
Angela Meader, CHCP
Jill A. Morris, PhD**
Joseph Muenzer, MD, PhD**
Dao Pan, PhD**
Lynda Polgreen, MD**
Uma Ramaswami, MD**
Mark S. Sands, PhD**
Dawn C. Saterdalen, RN, MBA
Ellen Sidransky, MD**
Patroula Smpokou, MD**
Ari Zimran, MD**
“WORLDSymposium 2021” 17th annual research meeting
WORLDSymposium™ is a licensed annual scientific research meeting, directed by Chester B. Whitley, PhD, MD, Course Director, and organized by an independent planning and organizing committee as listed above.
WORLD is the acronym for We’re Organizing Research on Lysosomal Diseases
The goal of WORLDSymposia is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. Each year, WORLDSymposia hosts a scientific meeting (WORLDSymposium) presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. WORLDSymposium is designed to help researchers and clinicians to better manage and understand diagnostic options for patients with lysosomal diseases, identify areas requiring additional basic and clinical research, public policy and regulatory attention, and identify the latest findings in the natural history of lysosomal diseases.
In all references and educational materials, it is WORLDSymposium’s editorial practice to eliminate the term “storage” in the context of “lysosomal storage disease”. This focuses attention on the growing body of knowledge indicating that ‘storage’ may not be the primary common mechanism of pathobiology in these conditions. While the physical accumulation of undegradable macromolecules might be important in disease manifestations like skeletal deformation, other processes such as inflammation and regulation of cell activity through the mTOR pathway, are increasingly recognized as more critical factors in the underlying pathology. To be clear, use of the term “lysosomal storage disease” (or “lysosomal storage disorder”) has changed to “lysosomal disease” (or “lysosomal disorder”) in all WORLDSymposium materials.
The lysosomal diseases are a collection of more than 40 clinical syndromes with incidence rates ranging from1 in 20,000 (Gaucher disease) to 1 in 300,000 (Wolman disease) live births; taken together these conditions are responsible for a significant amount of disability and disease burden. The rarity of each lysosomal disease means that no single medical research center has an opportunity to see sufficient numbers of patients with any one disease to effectively describe the full spectrum of each disease or adequately test any new therapies. The combined and integrated efforts of a network of centers with expertise in one or more of these diseases in order to solve major challenges in diagnosis, disease management, and therapy create solutions that will have a direct impact on patients suffering from lysosomal disease and will have important implications for medical practice.
Aspartylglucosaminuria; Batten disease; cholesteryl ester storage disease; Fabry disease; fucosidosis; galactosialidosis types I & II; Krabbe disease; neuronal ceroid lipofuscinosis (infantile, late infantile, juvenile); Mucolipidosis types II, III, & IV; Mucopolysaccharidosis types I, II, III, IV, and VI (Hurler, Hurler–Scheie, and Scheie; Hunter, Sanfilippo, Morquio, and Maroteaux–Lamy syndromes, respectively); Niemann–Pick disease; Pompe disease; Sandhoff disease; Schindler disease; sialidosis types I & II; Tay-Sachs disease; Wolman disease; alpha-mannosidosis types I & II; beta-mannosidosis.