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Article History

Received: 15 July 2025

Accepted: 29 October 2025

First Online: 24 November 2025

Declarations

:

: The Global HPP Registry study protocol was approved by the institutional review board or local equivalent at all participating study sites. Data collection and analysis was conducted in accordance with International Conference on Harmonisation Good Clinical Practice Guidelines and the Declaration of Helsinki. All patients provided written informed consent prior to registry enrollment.

: Not applicable.

: Priya S. Kishnani consults for and has received research funding and honoraria from Alexion, AstraZeneca Rare Disease. Lothar Seefried is a clinical study investigator and has received consultancy fees and institutional research funding and/or grant support from Alexion, AstraZeneca Rare Disease; Amgen; AM-Pharma; BioMarin; Chiesi; Haleon/GSK; Inozyme; Ipsen; Kyowa Kirin; MediPharma; Novartis; STADApharm; Theramex; UCB; and Ultragenyx. Keiichi Ozono has received research funding and honoraria, and support for meetings and/or travel from Alexion, AstraZeneca Rare Disease. Gabriel Ángel Martos-Moreno has consulted for and received research funding and honoraria from Alexion, AstraZeneca Rare Disease. Cheryl Rockman-Greenberg has consulted for and received research funding and honoraria from Alexion, AstraZeneca Rare Disease. Deborah Fowler is the chairman of the board of Soft Bones. Luke K. Burke, William R. Mowrey, Shona Fang, and Anna Petryk are employees of Alexion, AstraZeneca Rare Disease, and may hold stock or stock options in AstraZeneca. Eric T. Rush has received research funding to his institution from Alexion, AstraZeneca Rare Disease and Ultragenyx. He has received consulting fees and payment or honoraria from Alexion, AstraZeneca Rare Disease, Ultragenyx, Inozyme, Ipsen, and Kyowa Kirin. He has received support for meetings and/or travel from Alexion, AstraZeneca Rare Disease and Kyowa Kirin and participates on a data safety monitoring or advisory board for Inozyme. Peter R. Ebeling, Kathryn M. Dahir, and Agnès Linglart have consulted for and received research funding and honoraria from Alexion, AstraZeneca Rare Disease. Wolfgang Högler has consulted for and received research funding and honoraria from Alexion, AstraZeneca Rare Disease and BioMarin. He has received support for meetings and/or travel from BioMarin, Novo Nordisk, Sandoz and Alexion, AstraZeneca Rare Disease.