2020 Emerging Trends in Lysosomal Biology & Lysosomal Diseases: State-of-the-art for Experts

Monday, February 10, 2020 from 8:00 – 11:30 AM

For the eighth consecutive year, WORLDSymposium™ began with “Emerging Trends” on Monday morning, February 10, 2020 at 8:00 AM. This 3.5-hour CME/CE course provided a state-of-the-art update for experts working in lysosomal biology and lysosomal diseases. This course was a summary of the latest research trends and other advances in the field.

Seasoned researchers provided a global review of the past years’ advances, a state-of-the-art overview of lysosome biology, diseases and therapies. This review evolves every year, providing a summary of the latest research trends, new knowledge, and other discoveries. The course was 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. This course was taught at the postgraduate level, e.g., those with a PhD, MD, PharmD, DDS, MS, MPH, etc.

The content provided comprehensive information on lysosomal diseases, but does not overlap or replace the scientific data being presented during WORLDSymposium2020. Registration Required.

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

8:00 AM
Introduction and Overview of Course
Chester B. Whitley, PhD, MD

8:10 AM
Lysosomal Disease Phenotypes
Chester B. Whitley, PhD, MD

8:30 AM
Lysosomal Function and Pathogenesis
Steven U. Walkley, DVM, PhD

9:00 AM
Remarkable Cases I
Marc C. Patterson, MD

9:30 AM

9:45 AM
Current Treatments for Lysosomal Diseases
Jeanine R. Jarnes, PharmD

10:05 AM
Newborn Screening
Amy Gaviglio, MS, LCGC

10:25 AM
Future Treatments for Lysosomal Diseases
Jeanine R. Jarnes, PharmD

10:45 AM
Remarkable Cases II
Marc C. Patterson, MD

11:05 AM
Patient Advocate’s Perspective on Experimental Therapies
Cara O’Neill, MD

11:25 AM
Closing Remarks
Chester B. Whitley, PhD, MD

11:30 AM

Invited Faculty

Chair: Chester B. Whitley, PhD, MD
Course Director
WORLDSymposium and “Emerging Trends: State-of-the-Art for Experts”
Professor, Department of Pediatrics, and
Experimental and Clinical Pharmacology
University of Minnesota
Principal Investigator, Lysosomal Disease Network
Minneapolis, MN, USA

Steven U. Walkley, DVM, PhD
Director, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center
Head, Sidney Weisner Laboratory of Genetic Neurological Disease
Departments of Neuroscience, Pathology and Neurology
Rose F. Kennedy Center
Albert Einstein College of Medicine
Bronx, NY, USA

Marc C. Patterson, MD, FRACP
Professor of Neurology, Pediatrics and Medical Genetics
Editor-in-Chief, Journal of Child Neurology and Child Neurology Open
Editor, Journal of Inherited Metabolic Disease and JIMD Reports
Mayo Clinic Children’s Center
Rochester, MN, USA

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

Amy Gaviglio, MS, LCGC
G2S Corporation
Newborn Screening and Molecular Biology Branch
Division of Laboratory Sciences, NCEH
Centers for Disease Control and Prevention
Minneapolis, MN

Cara O’Neill, MD
Chief Science Officer
Cure Sanfilippo Foundation
Columbia, SC