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Article History

Received: 18 March 2025

Accepted: 22 November 2025

First Online: 20 January 2026

Declarations

:

: This study protocol was approved by the Emory IRB as IRB00072967. Informed consent to participate in the study was obtained from participants (or their parent or legal guardian in the case of children under 16). See Laney et al., 2024 for detailed methods.

: Not applicable.

: Anika Quillin, Hannah Waddel and Jared Druss do not have any competing interests to declare. Gwendolyn Gunn, PhD, MS, CGC, has disclosed that she has received honoraria and research funding as a speaker and registries/research coordinator for Sanofi Genzyme. These activities are monitored and in compliance with conflict of interest policies at Emory University. Nadia Ali has disclosed that she has received research support from Sanofi Genzyme, Shire Takeda, BioMarin, Amicus, and Pfizer, as well as lecturers’ honoraria from Sanofi Genzyme, BioMarin, Amicus, and Vitaflo. These activities are monitored and in compliance with conflict of interest policies at Emory University. William Wilcox, MD has disclosed research support from AmicusTherapeutics, clinical study contracts with Amicus, Chiesi, Protalix, Sangamo, Sanofi-Genzyme, and 4D Molecular Therapeutics, advisory meeting honoraria from Chiesi and Sanofi-Genzyme, and consultant involvement with Uniqure and Spark Therapeutics. Dawn Jacob Laney, MS, CGC, CCRC, has disclosed that she has received honoraria and research funding as an investigator, researcher, speaker, and board member registries/coordinator for Sanofi Genzyme; has received research funding and honoraria as an investigator, speaker, and coordinator for Amicus Therapeutics, Chiesi Therapeutics, and Shire (now part of Takeda); and is a co-founder and has stock options for ThinkGenetic, Inc.