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Article History

Received: 12 March 2024

Accepted: 13 October 2024

First Online: 28 March 2025

Declarations

:

: FOS and GOS registries have been approved by the ethics institutional review boards of participating centers and are conducted in compliance with relevant global and local regulations and best practices, Good Pharmacoepidemiological Practice, Good Research for Comparative Effectiveness principles, and the relevant principles of the International Conference on Harmonisation Good Clinical Practice guidelines (ICH E6), as appropriate for an observational registry. All participants provided written informed consent.

: Not applicable.

: SRV received grant/research support, honoraria, and advisory fees from Pfizer, Sanofi/Genzyme and Takeda. UR received honoraria for lectures and advisory boards from Amicus, Sanofi and Takeda; research grants from Amicus, Intrabio and Takeda. GPM received honoraria as a speaker and/or consultancy from Amicus, Chiesi, Lucane, and Takeda and unrestricted grants for research of Rare Diseases at VHIR from Sanofi and Takeda. DH received honoraria for speaking and advisory boards from Amicus, Chiesi, Freeline, Idorsia, Sangamo, Sanofi and Takeda. KN reports honoraria from Amicus Therapeutics, Sanofi Genzyme, and Takeda; and is a member of the FOS Steering Committee. RR reports honoraria, speaker fees, and consulting fees from Amicus Therapeutics, CSL Behring, Gador, Sanofi Genzyme, Novartis, and Takeda; and is a member of the FOS Steering Committee. RG received research grants, investigator fees, speaker/consultancy honoraria, and travel support to attend scientific meetings from Allievex, Amicus, Avrobio, Asafaros, BioMarin, Chiesi, Cyclo, DASA, Idorsia, Inventiva, Janssen, JCR, Lysogene, Novartis, Paradigm, PassageBio, Pfizer, Protalix, PTC, RegenxBio, Sanofi, Sigilon, Sobi,Takeda and Ultragenyx. OGA has received grant support from 4DMT, Chiesi, Idorsia, Protalix, Sangamo, and Sanofi for clinical research trials and from Sanofi and Takeda for other investigator-initiated studies; consulting fees from Chiesi, Sanofi, Takeda, and Uniqure; honoraria for speaker bureaus from Sanofi and Takeda and participates on the Advisory Board on Fabry Disease for Chiese and Sanofi. MI received support for the SZMC Gaucher Unit from Sanofi/Genzyme for participation in the ICGG Registry, from Takeda for the GOS Registry, and Pfizer for TALIAS; the Unit also receives research grants from Centogene, Pfizer, Sanofi/Genzyme and Takeda. AG was an employee of Takeda and stockholder in Takeda at the time of study start. MS has received honoraria and travel support from Alexion, Bi-938 oMarin, CHIESI, Orchard, Regenexbio, Sanofi, Takeda, and Ultragenyx. JB is an employee of Takeda and holds stock options in Takeda.