Document is current

Article History

Received: 7 January 2025

Accepted: 3 March 2025

First Online: 26 March 2025

Declarations

:

: Katie E. Moss reports support for advisory board participation/presentations from Alexion, AstraZeneca Rare Disease and receipt of educational grants from AbbVie; Amgen; Alexion, AstraZeneca Rare Disease; Novartis; and UCB. Richard Keen reports support for advisory board participation/presentations from Alexion, AstraZeneca Rare Disease. Alexandros Zygouras and Shona Fang are employees of Alexion, AstraZeneca Rare Disease and may own stock/options with AstraZeneca. Muhammad Javaid reports speaker honoraria and grants from Amgen, Kyowa Kirin, UCB, AbbVie, Besins Healthcare, and Sanofi. Tarekegn Geberhiwot and Peter Selby report no conflicts. Judith S. Bubbear reports support for speaker/consultancy participation from Alexion, AstraZeneca Rare Disease. Kenneth E.S. Poole reports support from the Cambridge NIHR Biomedical Research Centre. Jennifer S. Walsh reports speaker's honoraria from Eli Lilly, drug donation for clinical studies from Eli Lilly, and consulting fees from Mereo BioPharma.

: This analysis used prospective data collected from participants enrolled in the MAA for asfotase alfa between November 20, 2017, and February 2, 2023. The project complied with all relevant data protection and privacy regulations, including the European Data Protection Act and/or institution/country-specific participant privacy requirements and was performed in accordance with the Declaration of Helsinki. All participants were informed of the use and disclosure of their data. All patients consented to participate in the MAA, including attending regular appointments and completing questionnaires, and to have their data entered into the MAA database.