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™ 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.

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

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 2021. 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

10:00 AM
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

11:30 AM
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

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

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
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

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 live 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:
The National Society of Genetic Counselors (NSGC) has authorized WORLDSymposia, LLC to offer up to 1.947 CEUs or 19.47 Category 1 contact hours for the activity 17th Annual WORLDSymposium 2021. The American Board of Genetic Counseling (ABGC) will accept CEUs earned at this program for the purposes of genetic counselor certification and recertification.

(3.0 CEUs for Emerging Trends and 16.47 CEUs for WORLDSymposium 2021)

Genetic Counselors will be required to complete the online Evaluation on CME University (www.cmeuniversity.com) in order to receive a certificate.  Instructions will be provided in the Educational CME materials. The online evaluation should be completed within 2 weeks post-conference. Please note, you are required to pay the NSGC Fee of $25 at the time of registration in order to obtain NSGC Credit; if you did not pay the fee, you may not receive the NSGC CEU Credits. Upon completion of the online Evaluation, you will receive a Certificate of Completion, however, the Certificate of Credit will come directly from NSGC, you MUST include your NSGC number when you complete the online evaluation. If you have any questions, email Angela Meader at: angela@nullworldsymposia.org.