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Article History

Received: 14 April 2022

Revised: 21 November 2022

Accepted: 29 November 2022

First Online: 5 January 2023

Competing interests

: This work has received support from Utragenyx for medical writing and figure creation. CVS declares that she has in the past received funding from Ultragenyx for conference presentations and honoraria for participating in their Scientific Working Group. CVS is also Scientific Advisor (an unpaid role) for the ERNDIM (European Research Network for evaluation and improvement of screening, Diagnosis and treatment of Inherited disorders of Metabolism). AF declares that his institution has received funding from Amicus and Sanofi-Genzyme. AF has also received honoraria for participation on advisory boards from Amicus. JV declares that he has in the past received research funding from Ultragenyx and Reneo Pharmaceuticals. JV has also received payment or honoraria from CheckRare CME, Excel CEM, PTCE-PER CME, Med-IQ CME and Horizon Pharmaceuticals CME. JV plays are leadership from in SIMD NAMA and holds stocks in American Gene Therapies. CAB declares that she has received honoraria in the past for participating in scientific working groups for Utragenyx and Alnylam, and is unpaid board member of ANPGM, the French Association of Genetic Molecular Practitioners. NG declares that her institution has been the recipient of grants for clinical trials from Sanofi-Genzyme, Takeda and Chiesi. NG has received support for attending scientific meetings from Ultragenyx, Sanofi-Genzyme and Chiesi, as well as honoraria for participation on data safety monitoring and advisory boards from Ultragenyx, Sanofi-Genzyme, Chiesi and Biomarin.