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Article History

Received: 26 September 2023

Revised: 19 February 2024

Accepted: 29 February 2024

First Online: 2 April 2024

Declarations

:

: The contents of this manuscript are solely the responsibility of the authors and do not necessarily represent the official views of the NIH. Dr. Makhani is a consultant for the Institute for Advanced Clinical Trials (I-ACT) for Children. Dr. Lebrun-Frenay has nothing to disclose. Dr. Siva received honoraria or consultancy fees for participating in advisory boards, giving educational lectures, and/or travel and registration coverage for attending scientific congresses or symposia from F. Hoffmann-La Roche Ltd, Novartis, Sanofi-Genzyme, Merck-Serono, Teva, Biogen Idec/Gen Pharma of Turkey, Alexion, and Abdi Ibrahim Ilaç, Turkey. Dr. Shabanova has nothing to disclose. Dr. Wassmer received educational travel grants, consultancy and/or speaking fees from UCB, Shire, Merck Serona, Novartis, Bayer, Biogen Idec, Genzyme, Novartis, PTC Therapeutics, Alexion, GMP-Orphan and IGES U.K. Pharma Ltd. Dr. Santoro has consulting relationships with UCB and Cycle Pharmaceuticals. Dr. Narula has nothing to disclose. Dr. Brenton has served as a consultant for Cycle Pharmaceuticals and I-ACT for Children on a Novartis-sponsored project. Dr. Mar has nothing to disclose. Dr. Durand Dubief has nothing to disclose. Dr Zephir received board consulting fees, speaker honoraria, and travel support for congresses from Biogen Idec, Sanofi, Merck, Novartis, BMS, Alexion, Horizon Therapeutics, Roche, Janssen-Cilag. Dr. Mathey had travel/accommodations/meeting expenses funded by Biogen, Novartis, Sanofi-Genzyme, Merck, Teva, and Roche. Dr. Rojas has nothing to disclose. Dr. de Seze has nothing to disclose. Dr. Tenembaum has nothing to disclose. Dr. Stone has nothing to disclose. Dr. Casez has relevant financial activities outside the submitted work: Biogen (personal fees), Roche (personal fees, nonfinancial support (traveling/congress), Merck (personal fees, nonfinancial support (traveling/congress), Novartis (personal fees, nonfinancial support (traveling/congress), Jansen (personal fees), and Sanofi (personal fees). Dr. Carra-Dalliere received travel grants and/or consulting fees from Alexion, Biogen, Merck, Novartis, Roche, and Sanofi-Genzyme. Dr. Neuteboom has nothing to disclose. Dr. Ahsan has nothing to disclose. Dr. Arroyo has nothing to disclose. Dr. Cabre has nothing to disclose. Dr. Gombolay receives part-time salary support from the CDC for acute flaccid myelitis case reviews and has received an honorarium from Pediatric Neurology as a Media Editor. Dr. Inglese has nothing to disclose. Dr. Louapre has received consulting or travel fees from Biogen, Novartis, Roche, Sanofi, Teva, and Merck Serono, and a research grant from Biogen. Dr. Margoni received personal fees from Almirall and Merck. Dr. Palavra has nothing to disclose. Dr. Pohl has nothing to disclose. Dr. Reich receives research support from Abata and Sanofi. Dr. Ruet reports outside the submitted work research grants to the institution from Biogen, BMS, Genzyme, Merck, Roche, and personal fees and/or non-financial support from Biogen, Alexion, Novartis, Horizon Th, and Merck. Dr. Thouvenot received consulting and lecturing fees, travel grants, or unconditional research support from the following pharmaceutical companies: Actelion, Biogen, BMS, Merck, Novartis, Roche, and Sanofi. Dr. Timby has nothing to disclose. Dr. Tintore has received compensation for consulting services, speaking honoraria, and research support from Almirall, Bayer Schering Pharma, Biogen-Idec, Genzyme, Janssen, Merck-Serono, Novartis, Roche, Sanofi-Aventis, Viela Bio and Teva Pharmaceuticals and is on a data Safety Monitoring Board for Parexel and UCB Biopharma. Dr. Azevedo has received consulting fees from Horizon Therapeutics, Genentech, Sanofi Genzyme, TG Therapeutics, and EMD Serono. Dr. Okuda received personal compensation for consulting and advisory services from Alexion, Biogen, Celgene/Bristol Myers Squibb, EMD Serono, Genentech, Genzyme, Janssen Pharmaceuticals, Osmotica Pharmaceuticals, RVL Pharmaceuticals, Inc., TG Therapeutics, and research support from Biogen, EMD Serono/Merck, and Novartis. Dr. Okuda has issued national and international patents along with pending patents related to other developed technologies. Dr. Okuda received royalties for intellectual property licensed by The Board of Regents of The University of Texas System. Drs. Uygunoglu, Vargas, Venkateswaran, Verhelst, Wickstrom, Kantarci, Shapiro, and Pelletier have nothing to disclose.

: Institutional ethical approvals were obtained at all sites. Written informed consent was obtained from parents/guardians and children provided assent.