1. Pediatrics, University of Utah, Salt Lake City, UT, USA
2. Institute of Translational Medicine, University of Liverpool, Liverpool, UK
3. Department of Clinical Sciences Lund, Pediatrics, Lund University, Skåne University Hospital, Lund, Sweden
4. Department of Pediatrics, Brody School of Medicine, East Carolina University, Greenville, NC, USA
5. Patient Centered Outcomes, ICON, South San Francisco, CA, USA
6. Patient Centered Outcomes, ICON, Gaithersburg, MD, USA
7. Patient Centered Outcomes, ICON, Oxford, UK
8. Global Clinical Development, Rare Metabolic Diseases, Takeda, Switzerland
9. Global Clinical Development, Rare Metabolic Diseases, Takeda, Lexington, MA, USA
10. Global Evidence and Outcomes, Takeda, Lexington, MA, USA