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Article History

Received: 14 April 2023

Accepted: 6 July 2023

First Online: 16 November 2023

Declarations

:

: The study was conducted in compliance with Good Clinical Practice as described in the Code of United States Federal Regulations, the International Council for Harmonisation of Good Clinical Practice Guidelines, and the ethical principles of the Declaration of Helsinki. The protocol and informed consent documents were reviewed and approved by the institutional review board and/or independent ethics committee at each site participating in this study. For all patients, written informed consent was obtained from their parent(s) or legally authorized guardian(s) before any study-related procedures were conducted.

: Not applicable.

: JM has received consulting fees/other remuneration from Denali Therapeutics, JCR Pharmaceuticals, REGENXBIO, Sangamo Therapeutics, Sanofi Genzyme, and Takeda; has participated in advisory boards for BioMarin Pharmaceutical, Denali Therapeutics, JCR Pharmaceuticals, Sanofi Genzyme, and Takeda; and has received research support from BioMarin Pharmaceutical, Denali Therapeutics, and Takeda.BKB has received consulting fees/other remuneration from Alexion Pharmaceuticals, Applied Therapeutics, BioMarin Pharmaceutical, Capsida Biotherapeutics, Denali Therapeutics, Horizon Therapeutics, JCR Pharmaceuticals, Moderna, Passage Bio, Sanofi Genzyme, Sio Gene Therapies, Takeda, and Ultragenyx Pharmaceutical; has participated in advisory boards or similar committees for Alexion Pharmaceuticals, BioMarin Pharmaceutical, and Takeda; and has received research support from Alexion Pharmaceuticals, BioMarin Pharmaceutical, Denali Therapeutics, Homology Medicines, JCR Pharmaceuticals, Sangamo Therapeutics, Takeda, and Ultragenyx Pharmaceutical.HMA has participated in advisory boards for Takeda; has received consulting fees from BioMarin Pharmaceutical and Takeda; has been involved in contracted research for Amicus Therapeutics, Bluebird bio, and Takeda; has received fees from Amicus Therapeutics, BioMarin Pharmaceutical, Sanofi Genzyme, and Takeda; and has received other remuneration from Amicus Therapeutics, Bluebird bio, and Takeda.PRH has received consulting fees/other remuneration from Aeglea, Alexion Pharmaceuticals, ArmaGen, Audentes, AVROBIO, BioMarin Pharmaceutical, Capsida Biotherapeutics, Chiesi, Denali Therapeutics, Edigene, Enzyvant, Fondazione Telethon, Grace Science, Inventiva Pharma, JCR Pharmaceuticals, Novel Pharma, Orchard Therapeutics, Orphazyme, Paradigm Biopharma, PTC Therapeutics, Rallybio, REGENXBIO, Renoviron, Saliogen, Sangamo Therapeutics, Sanofi Genzyme, Takeda, and Ultragenyx Pharmaceutical; and has received research support from Adrenas Therapeutics, Amicus Therapeutics, Alexion Pharmaceuticals, ArmaGen, Ascendis Pharma, Aspa Therapeutics, Azafaros, BioMarin Pharmaceutical, Calcilytix, Denali Therapeutics, Enzyvant, Homology Medicines, Inventiva Pharma, JCR Pharmaceuticals, Orphazyme, Prevail Therapeutics, REGENXBIO, Sangamo Therapeutics, Swedish Orphan Biovitrum, Takeda, and Ultragenyx Pharmaceutical.LGG-S has received consulting fees/other remuneration from BioMarin Pharmaceutical, Sanofi Genzyme, Takeda, and Ultragenyx Pharmaceutical; and has received research support from Takeda.MR-G has received consulting fees/other remuneration and research support from Takeda.YW is an employee of Takeda Development Center Americas, Inc. and is a stockholder of Takeda Pharmaceuticals Company Limited.DM is an employee of Takeda Development Center Americas, Inc. and is a stockholder of Takeda Pharmaceuticals Company Limited.DA was an employee of Takeda Development Center Americas, Inc. at the time of this study and of the writing of the manuscript (now with Merck) and is a stockholder of Takeda Pharmaceuticals Company Limited.SAJ has received consulting fees/other remuneration from Alexion Pharmaceuticals, AVROBIO, BioMarin Pharmaceutical, Denali Therapeutics, Orchard Therapeutics, REGENXBIO, Sanofi Genzyme, Takeda, and Ultragenyx Pharmaceutical; and has received research support from Avrobio, Orchard Therapeutics, and Takeda.