2015 Emerging Trends in Lysosomal Biology & Lysosomal Diseases: State-of-the-art for Experts

Monday, February 9, 2015 from 1:00 – 5:00 PM

The Emerging Trends 2015 pre-conference session provided a foundation for researchers and health care practitioners interested in gaining more background on the diagnosis and treatment of lysosomal diseases. Content covered accepted methods of diagnosis and treatment of the known lysosomal diseases. New and/or ongoing research was not included in this activity. Emerging Trends 2015 was taught at the graduate-school level and was targeted towards people who wanted to learn more of the fundamental information that provides the basis for future research and treatments, as well as those who would benefit from a refresher on this topic.

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.


1:00 PM
Introduction and Overview, ARS Demo Questions
Chester B. Whitley, PhD, MD

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

2:00 PM
Lysosomal Diseases and Pathology
Chester B. Whitley, PhD, MD

2:55 PM

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

4:00 PM
Clinical Correlation: Cases of Lysosomal Disease
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