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™ will begin 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. This course is a summary of the latest research trends and other advances in the field.

Seasoned researchers provide 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 2022.

Emerging Trends 2022 will be offered as a  pre-recorded virtual-only session that will be 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.)

Registration Required: $75 if registered on or before January 8, 2022; $85 if registered after January 8, 2022.

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

Introduction
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


Target Audience:
This activity has been designed for geneticists, pediatricians, primary care practitioners, registered nurses, nurse practitioners, physician assistants, genetic counselors, and other researchers and health care practitioners involved with the identification of disease mechanisms and potential treatments, or involved with the diagnosis and management of individuals with lysosomal diseases. In addition, the content is designed to be applicable to individuals with lysosomal diseases and their family members, as well as advocacy groups involved with raising awareness.

Joint Accreditation Statement:
In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and WORLDSymposia. Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Physician Continuing Medical Education:
The Postgraduate Institute for Medicine designates this enduring activity for a maximum of 3.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Continuing Nursing Education:
The maximum number of hours awarded for this Continuing Nursing Education activity is 3.0 contact hours.

Genetic Counselor CEUs:
This event will be submitted to the National Society of Genetic Counselors (NSGC) for approval of Category 1 CEUs. The American Board of Genetic Counseling (ABGC) accepts CEUs approved by NSGC for purposes of recertification. Approval for the requested 3.000 Contact Hours is currently pending.