Update on Therapeutics in Inborn Errors

December 15, 2023

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: 
12/15/2023 - 12:00pm MST
Event ends: 
12/15/2023 - 1:30pm MST
Children's Hospital Colorado - Virtual
Aurora, CO 80045
United States

Guest Faculty

Cary Harding, MD
Oregon Health & Science University


Cary Harding, speaker for this activity, is a consultant for BioMarin Pharmaceutical, Ultragenyx Pharmaceutical, Synlogic Therapeutics, Sanofi-Genzyme, Jnana Therapeutics, PTC Therapeutics and American Gene Technologies, and a Data Safety Monitoring Board Member for LogicBio 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


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