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

Monday, January 31, 2022 – March 15, 2022

For the tenth consecutive year, WORLDSymposium™ began with “Emerging Trends”. This half day CME/CE course provides a state-of-the-art update for experts working in lysosomal biology and lysosomal diseases.
Emerging Trends 2022 was offered as a  pre-recorded virtual-only session that was available to view online starting Monday, January 31, 2022  and available to watch on demand until March 15, 2022.  (No in-person meeting will be held for this session, it will be virtual online only.)

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.
  5. Identify important regulatory considerations in the design of a clinical trial for lysosomal diseases.

Preliminary Agenda

Chester B. Whitley, PhD, MD
Lysosomal Function and Pathogenesis
Andrew P. Lieberman, PhD, MD
Clinical Features
Marc C. Patterson, MD
Newborn Screening
Amy Gaviglio, MS, LCGC
Lysosomal Disease Therapies
Jeanine R. Jarnes, PharmD
Regulatory Review
Patroula Smpokou, MD
COVID-19 Fireside Discussion
Michael Osterholm, PhD, MPH
Case Studies
Marc C. Patterson, MD
Parent/Patient’s Perspective: International
Alan Finglas
Parent’s Perspective on Rare Disease Drug Development: US
Melissa Hogan, JD
Closing Remarks
Chester B. Whitley, PhD, MD

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, MI, 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
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, USA
Jeanine R. Jarnes, PharmD, BCOP, BCPS
Assistant Professor
Department of Pediatrics, Medical School and
Experimental and Clinical Pharmacology
College of Pharmacy
University of Minnesota
Minneapolis, MN, USA
Patroula Smpokou, MD
Deputy Director
Division of Rare Diseases & Medical Genetics
Office of New Drugs | CDER | FDA
Silver Spring, MD, USA
Michael Osterholm, PhD, MPH
Regents Professor
McKnight Presidential Endowed Chair in Public Health
Director, Center for Infectious Disease Research and Policy
Distinguished Teaching Professor, Division of Environmental Health Sciences
School of Public Health
Professor, Technological Leadership Institute, College of Science & Engineering
Adjunct Professor, Medical School
University of Minnesota
Minneapolis, MN, USA
Alan Finglas
MSD Action Foundation / SavingDylan.com
Founder & Research Manager
Dublin, Ireland
Patient Advocate and Parent/Caregiver to a child affected by
Multiple Sulfatase Deficiency
Melissa J. Hogan, JD
Principal Consultant
Doulots, LLC
Inaugural Member, Patient Engagement Collaborative
U.S. Food and Drug Administration
Author, “Afraid of the Doctor: Every Parent’s Guide to Preventing and Managing Medical Trauma”
Founder, Project Alive, a Hunter Syndrome Research and Advocacy Foundation
Nashville, TN, USA