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Article History

Received: 13 January 2025

Accepted: 11 July 2025

First Online: 13 August 2025

Declarations

:

: Not applicable.

: Not applicable.

: RJH is a member of the Fabry Registry Advisory Board and the FollowMe registry, consults with Sanofi and Amicus Therapeutics, Chiesi Farmaceutici and Freeline, and has been an investigator in clinical trials sponsored by Amicus Therapeutics, Idorsia, Protalix Biotherapeutics, Sangamo Therapeutics, Sanofi, and Takeda. These activities have been monitored and found to be in compliance with the conflict of interest policies at Cincinnati Children’s Hospital Medical Center. DL is chair of the North American Fabry Registry Board, consults and/or has been an investigator and coordinator in clinical trials sponsored by 4DMT, Amicus Therapeutics, Chiesi Farmaceutici, Protalix BioTherapeutics, Sangamo, Sanofi-Genzyme, Spark Therapeutics, and Takeda-Shire. These activities have been monitored and found to be in compliance with the conflict of interest policies at the Emory University School of Medicine. AW and SK are employees and shareholders of Sanofi.

: Some data within the manuscript were presented at the 18th Annual WORLD Symposium hybrid (virtual and in-person) congress, February 7–11, 2022.