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

Tuesday, February 21, 2023, 4:00 – 6:00 PM EST

For the eleventh consecutive year, WORLDSymposium™ began with “Emerging Trends”. This 2-hour 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 2023.

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.

2023 Emerging Trends Agenda

Chester B. Whitley, PhD, MD

Lysosomal Function and Pathogenesis
Gregory A. Grabowski, MD

Clinical Features
Marc C. Patterson, MD

Newborn Screening
Amy Gaviglio, MS, LCGC

Lysosomal Disease Therapies
Jeanine R. Jarnes, PharmD

Regulatory Review
Christine Yuen-Yi Hon, PharmD

Patient Perspective
Jennifer Klein, MS 

Rare Disease Research
N. Matthew Ellinwood, DVM, PhD 

Panel Q&A
All Speakers

2023 Emerging Trends 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

Gregory A. Grabowski, MD
Professor Emeritus 
University of Cincinnati College of Medicine 
Departments of Pediatrics, and Molecular Genetics,  
Biochemistry and Microbiology 
Division of Human Genetics 
Cincinnati Children’s Hospital Research Foundation 
Cincinnati, OH, 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
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, USA

Christine Yuen-Yi Hon, PharmD
Clinical Analyst
Division of Rare Diseases & Medical Genetics
Office of New Drugs | CDER | FDA
Silver Spring, MD, USA

Jennifer Klein, MS
Operations and Program Management
Odylia Therapeutics
Scientific Advisory Board, ISMRD
Co-Founder, Mucolipidosis Collaborative Research Network 
Adult Resource Committee 
National MPS Society 
Durham, NC, USA 

N. Matthew Ellinwood, DVM, PhD
Chief Scientific Officer 
National MPS Society 
Durham, NC, 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.

Jointly Accredited Provider Logo

Joint Accreditation Statement:
In support of improving patient care, this activity has been planned and implemented by Partners for Advancing Clinical Education (PACE) and WORLDSymposium. PACE 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 Education:
PACE designates this activity for a maximum of 2.0 AMA PRA Category 1 Credit(s).  Physicians should claim only the credit commensurate with the extent of their participation in the activity. 

Nursing Continuing Education:
The maximum number of hours awarded for this Continuing Nursing Education is 2.0 contact hours. 

Genetic Counselor CEUs:
The National Society of Genetic Counselors (NSGC) has authorized WORLDSymposia, LLC, to offer up to 2.583 CEUs or 25.83 Category 1 contact hours for the activity 19th Annual WORLDSymposium 2023. The American Board of Genetic Counseling (ABGC) will accept CEUs earned at this program for the purposes of genetic counselor certification and recertification. 

  • WORLDSymposium – 2.583 CEUS or 25.83 Category 1 contact hours 
  • Emerging Trends – 0.200 CEUs or 2.00 Category 1 contact hours