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Article History

Received: 21 December 2022

Accepted: 24 August 2023

First Online: 27 September 2023

Declarations

:

: This registry is run in accordance with the recommendations from the Declaration of Helsinki and has received ethical, regulatory and institutional approvals at national, regional and site level for each participating country, as required. All participants or their legal representative provide informed consent before their enrolment in the registry.

: Not applicable.

: GA has received honoraria for consultancy and for lecturing in educational activities from Kyowa Kirin International. SSBN has received honoraria for providing consultancy from Kyowa Kirin International; and research grants, in addition to honoraria, for providing consultancy from Inozyme Pharma. AMB has received research grants and honoraria for providing consultancy and lectures from Kyowa Kirin International. MLB has received honoraria from Amgen, Bruno Farmaceutici S.p.A., Calcilytix, Kyowa Kirin International and UCB Pharma; grants and/or speaker fees from Abiogen Pharma, Alexion, Amgen, Bruno Farmaceutici S.p.A., Echolight, Eli Lilly and Company, Kyowa Kirin International, SPA, Theramex and UCB Pharma; and consultancy fees from Alexion, Amolyt Pharma, Bruno Farmaceutici S.p.A., Calcilytix, Kyowa Kirin International and UCB Pharma. KB has received honoraria as a consultant from Amgen, Kyowa Kirin International, Theramex and UCB Pharma. CLC has received honoraria for consultancy and for lecturing in educational activities from Kyowa Kirin International. FE has received honoraria from Chiesi Farmaceutici, Kyowa Kirin International and Recordati Rare Diseases; grants and/or speaker fees from Chiesi Farmaceutici, Kyowa Kirin International and Recordati Rare Diseases; and consultant fees from Alnylam, AVROBIO, Kyowa Kirin International and Otsuka. SG has no competing interests to declare. DH has received consultancy and speaker fees from Chiesi Farmaceutici and Kyowa Kirin International; and research grants from Amgen, Chiesi Farmaceutici and Kyowa Kirin International. RK has received honoraria from Kyowa Kirin International for advisory board participation. EL has received consultancy fees from Kyowa Kirin International. OM has received honoraria as a consultant from BridgeBio, Kyowa Kirin International and Ultragenyx. MZM has received honoraria for attending advisory boards and for educational lectures/webinars from Kyowa Kirin International. ON has received consultancy honoraria and research funding from Kyowa Kirin International. DS serves as a consultant for Kyowa Kirin Germany; and has received honoraria for attending advisory boards from Kyowa Kirin International. LTS has received honoraria as a consultant from Amgen and Kyowa Kirin International. JL and AW are employees of Kyowa Kirin International. SW was an employee of Kyowa Kirin International at the time of drafting this manuscript. MCZ reports that her institution has received a research grant from Kyowa Kirin International.