Update on Therapeutics in Inborn Errors

October 25, 2024

The Update on Therapeutics in Inborn Errors session will discuss updates and discussion of novel therapies including gene therapy trials and techniques.

Commercial Supporter 

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

Learning Objectives

  • Better understand the driver’s of therapeutic development on inborn errors of metabolism
  • Appreciate the role of the Orphan Drug Act on rare disease treatments
  • Recognize ways to be involved in drug development for rare IEMs
Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Attendance
Course opens: 
Course expires: 
Event starts: 
10/25/2024 - 12:00pm MDT
Event ends: 
10/25/2024 - 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

Shawn McCandless, MD
Children's Hospital Colorado


Shawn McCandless, MD, speaker for this activity, is a consultant for Levo Therapeutics, a speaker for Horizon Therapeutics and a researcher for Aardvark Therapeutics, Ultragenyx and Harmony Therapeutics. 

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.