Late and Attenuated Presentations in Lysosomal Storage Disorders
This CME workshop will focus on basic mechanisms, varied clinical phenotypes, and establishing goals for disease monitoring and various treatment strategies for late onset/attenuated types of LSDs.
LSDs in focus
· Fabry disease
· Gaucher disease
· Pompe disease
· Tay-Sachs disease
· Mucopolysaccharidoses (MPS)
· Niemann-Pick Type C1
Kazuya Tsuboi, M.D.
Director, LSD Center, Nagoya Central Hospital, Japan
Shoshana Revel-Vilk, M.D.
Gaucher Unit, Pediatric Hematology Unit Shaare Zedek Medical Center, Jerusalem, Israel
Mia Horowitz, Ph.D
Dept. Cell Research and Immunology, Tel Aviv University,Ramat Aviv, Israel
Nina Raben, M.D., Ph.D
Division of Muscle Biology, National Institutes of Health, Maryland, USA
Orna Staretz-Chacham, M.D.
Senior Neonatologist, Metabolic Clinic at Soroka Medical Centre Beersheva, Israel
Raphael Schiffmann, M.D., M.H.Sc., FAAN
Director, Institute of Metabolic Disease, Baylor Research Institute Texas, USA
Deborah Elstein , Ph.D
Silvia Muro, Ph.D
Associate Professor of Bioengineering, University of Maryland, USA
Uma Ramaswami FRCPCH, M.D.
Consultant in Inherited Metabolic Disorders Clinical Lead, Lysosomal Disorders Unit
Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust
Pond Street, London
Pranoot Tanpaiboon, M.D., FACMGG
Associate Medical Director, Advanced Diagnostics - Genetics, Genomics and R&D, Biochemical Genetics , Quest Diagnostics , San Juan Capistrano, CA , USA
Ibrahim T. Ozbolat, Ph.D
Harzt Family Associate Professor , Penn State University , PA, USA
Hanna Rosenbaum M.D.
Head of Hematology clinic, Hematology Day care unit and Center for Gaucher disease. The center for Consultant Medicine, Clalit, Nazareth, Israel
Vice President High Throughput Testing & Biomarker Development, Centogene, Germany
Swati Sathe, M.D., MS
Director of Neurogenetics Division, Director Inpatient Neurology, Northwell Health, Staten Island University Hospital, USA
Ellen Sidransky, M.D.
Chief, Section on Molecular Neurogenetics MGB, National Human Genome Research Institute, USA
James Shayman, M.D.
Professor, Internal Medicine, University of Michigan, USA
Heather A. Lau, M.D. M.S.
Assistant Professor, Child Neurology, Director, Lysosomal Storage Disorders Program
Neurogenetics Division Department of Neurology,New York University School of Medicine, USA
Asa Abeliovich, M.D., Ph.D
Founder & CEO of Prevail Therapeutics, USA