2017 Emerging Trends in Lysosomal Biology & Lysosomal Diseases: State-of-the-art for Experts

Monday, February 13, 2017 from 1:00 – 5:00 PM

For the fifth consecutive year, WORLDSymposium™ began with “Emerging Trends” on Monday afternoon, February 13, 2017 at 1:00 PM. This 4-hour CME course provides a state-of-the-art update for experts working in lysosomal biology and lysosomal diseases. This course is a summary of the latest research trends and other advances in the field. The course is intended for researchers and health care practitioners who are interested in being current on recent advances in the basic science, diagnosis, and treatment of lysosomal diseases. Presentations are at the postgraduate level, e.g., those with a PhD, MD, PharmD, DDS, MS, MPH, etc. The content provides comprehensive information on lysosomal diseases, but does not overlap or replace the scientific data being presented during WORLDSymposium 2017.

Learning Objectives

Upon completion of this educational activity, the participant should be better able to:

  1. Describe the basic structure, function and molecular biology of lysosomes.
  2. Identify specific lysosomal diseases, their clinical manifestations, and means of diagnosis.
  3. Review current treatments for lysosomal diseases, the potential side effects, and their expected clinical outcomes.
  4. Correlate the molecular biology of lysosomes with clinical features, diagnostic testing, and treatment approaches.

Preliminary Agenda

1:00 PM
Welcome: Rationale and Course Goals
Chester B. Whitley, PhD, MD

1:10 PM
Molecular Biology and Cell Biology of Lysosomes
Steven U. Walkley, DVM, PhD

2:00 PM
Lysosomal Testing and Outcome Measures
Chester B. Whitley, PhD, MD

2:55 PM

3:10 PM
Treatments for Lysosomal Diseases
Jeanine R. Utz, PharmD

4:00 PM
Remarkable Cases
Marc C. Patterson, MD

4:55 PM
Final Q&A and Evaluation

5:00 PM

Invited Faculty

Chair: Chester B. Whitley, PhD, MD
Principal Investigator, Lysosomal Disease Network
Professor of Pediatrics,
Department of Pediatrics,
University of Minnesota,
Minneapolis, MN, USA

Marc C. Patterson, MD, FRACP
Chair – Division of Child and Adolescent Neurology
Professor of Neurology, Pediatrics and Medical Genetics
Director of the Child Neurology Training Program,
Mayo Clinic Children’s Center
Rochester, MN, USA

Jeanine R. Utz, PharmD, BCOP, BCPS
Adjunct Assistant Professor, Experimental and Clinical Pharmacology
College of Pharmacy
University of Minnesota, Fairview
Minneapolis, MN, USA

Steven U. Walkley, DVM, PhD
Professor, Dominick P. Purpura Department of Neuroscience
Professor, Department of Pathology
Professor, The Saul R. Korey Department of Neurology
Director, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center,
Albert Einstein College of Medicine
Rose F. Kennedy Center
Bronx, NY, USA