Lysosomal Disorders 2

October 18, 2024

The Lysosomal Disorders 2 session will be discussing Sphingolipid/ceramide disorders (Fabry, Gaucher, Krabbe, etc.), Neuronal Ceroid Lipofuscinoses.

Commercial Supporter 

This educational activity is supported by an educational grant from Ultragenyx.

Learning Objectives

  • Recognize symptoms and signs of a lysosomal storage disease predominantly affecting the brain. 
  • Understand how the prevalence of pseudodeficiencies impact newborn screening and diagnostic testing. 
  • Become familiar with new and emerging therapies for the neurological lysosomal storage disease. 
Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Attendance
Course opens: 
Course expires: 
Event starts: 
10/18/2024 - 12:00pm MDT
Event ends: 
10/18/2024 - 1:30pm MDT
Children's Hospital Colorado
Aurora, CO 80045
United States

Medical Biochemical Genetics is hosted virtually via Zoom. Zoom details will be provided after registering for the course. 

Guest Faculty

William Wilcox, MD, PhD
Emory University


William Wilcox, MD, PhD, speaker for this activity, is on the advisory board for Sanofi-Genzyme, Chiesi, Takeda and Homology Medicines, is a principle investigator on contracted clinical research for Amicus Therapeutics, BioMarin, Protalix, Chiesi, Takeda, Alexion/AstraZeneca, 4D Molecular Therapeutics, Sangamo, Astellas, Orphazyme and Pfizer, is a consultant for Spark Therapeutics/Roche and UniQure, and has received a research grant from Amicus Therapeutics and Takeda. 

Planners, faculty, and others in control of content (either individually or as a group) have no relevant financial relationships with ineligible companies.

All of the relevant financial relationships listed have been mitigated. 


Children’s Hospital Colorado is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

Children’s Hospital Colorado designates this Live activity for a maximum of 1.50 AMA PRA Category 1 Credit(s)™. Physicians should only claim credit commensurate with the extent of their participation in the activity.


A general certificate of attendance will be available after the completion of the online evaluation. Claim only credit commensurate of your conference attendance.

Available Credit

  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Attendance


Please login or register to take this course.