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Article History

Received: 27 January 2025

Accepted: 25 April 2025

First Online: 29 May 2025

Declarations

:

: Raja Padidela: Research grant and honoraria from Alexion, AstraZeneca Rare Disease, Ultragenyx-Mereo, and Bridgebio. Nick Bishop: Investigator for Alexion, AstraZeneca Rare Disease; UK CI for Amgen; CI for Ultragenyx-Mereo; consultant for Amgen, Novo Nordisk, and Rampart; consultant and advisory board participant for Alexion, AstraZeneca Rare Disease, Ultragenyx, and Mereo; research grant from Horizon Europe. Paul Arundel: Principal investigator and advisory board participant with and recipient of honoraria from Alexion, AstraZeneca Rare Disease. Shona Fang and Alexandros Zygouras: Employees of and may own stock/options in Alexion, AstraZeneca Rare Disease. M. Zulf Mughal: Recipient of honoraria from Alexion, AstraZeneca Rare Disease and Biologix. Nick Shaw: Nothing to disclose. Vrinda Saraff: Investigator for and recipient of honoraria from Alexion, AstraZeneca Rare Disease.

: Data were collected prospectively from all participants enrolled in the MAA in the UK. All enrolled participants (or their legally authorized representative) were informed of the purpose of the data collection project and the expected use of any data collected. 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. The project was conducted in accordance with the Declaration of Helsinki. All participants consented to participate in the MAA, including completing questionnaires and attending appointments, and to have their data entered into the MAA database. The program was started in August 2017 and concluded in February 2023.