Emerging Trends in Lysosomal Biology & Lysosomal Diseases: STATE-OF-THE-ART FOR EXPERTS

Sunday, February 7, 2021 – March 15, 2021: On-Demand Only

For the ninth consecutive year, WORLDSymposium™ began with “Emerging Trends”. This half day 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.

Emerging Trends was a pre-recorded virtual-only session that was available to view online starting Sunday, February 7, 2021 at 10:00 AM Eastern Standard Time (EST). After the session concluded at 2:00 PM Eastern Standard Time (EST), it was available to watch on-demand until March 15, 2021.

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 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. This course is taught 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 2021. Registration was 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

10:00 AM
Introduction
Chester B. Whitley, PhD, MD

10:05 AM
Lysosomal Function and Pathogenesis
Andrew P. Lieberman, PhD MD

10:25 AM
Clinical Features
Marc C. Patterson, MD

10:45 AM
Newborn Screening
Amy Gaviglio, MS, LCGC

11:05 AM
Break

11:30 AM
Therapies
Jeanine R. Jarnes, PharmD

11:50 AM
FDA Regulatory Review
Patroula Smpokou, MD

12:10 PM
Patient’s Perspective on Experimental Therapies
Cara O’Neill, MD

12:30 PM
Break

1:00 PM
Case Studies
Marc C. Patterson, MD

1:20 PM
SARS-CoV-2 Virus
Chester B. Whitley, PhD, MD

1:40 PM
COVID-19: Clinical Impact and Management
Heather Lau, MD

2:00 PM
Closing Remarks
Chester B. Whitley, PhD, MD

2:05 PM
Adjourn

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

Andrew P. Lieberman, PhD MD
Abrams Collegiate Professor of Pathology
Director of Neuropathology
University of Michigan Medical School
Ann Arbor, Michigan

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

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

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

Patroula Smpokou, MD
Deputy Director
Division of Rare Diseases & Medical Genetics
Office of New Drugs │ CDER | FDA
Silver Spring MD

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

Heather Lau, MD
Assistant Professor, Department of Neurology
Associate Director, Division of Neurogenetics
Director of the Lysosomal Storage Disorder Program
New York University School of Medicine
Neurogenetics Division
New York, NY