Document is current

Article History

Received: 6 October 2025

Accepted: 26 January 2026

First Online: 24 March 2026

Declarations

:

: Florence Lacaille, Vlad Ratziu, and Gérard de Pouvourville received consultancy fees from Alexion, AstraZeneca Rare Disease. François Feillet reported receiving a grant from Recordati to support a PhD thesis, consultancy fees from Jana Therapeutics, PTC Therapeutics, Vitaflo, Danone, and Alexion, AstraZeneca Rare Disease, honoraria for lectures, manuscript writing, or educational events from Recordati and Alexion, AstraZeneca Rare Disease, and support for attending meetings and advisory board participation from PTC Therapeutics. Ekbel Amri and Florian Abel (FA) are employees of Alexion, AstraZeneca and may hold stock or stock options in AstraZeneca. Helène Chevrou-Severac was an employee at Alexion, AstraZeneca Rare Disease during project and manuscript development. Sophie Marguet, Vanessa Rascon Velasco, and Reyhan Hasanova are employees of Oracle France, which received fees from Alexion, AstraZeneca Rare Disease to conduct the analysis and develop the manuscript.

: Our study involved anonymized structured data that does not include any personally identifiable information and, as such, it is not governed by privacy laws requiring individual patient consent, in accordance with relevant regulatory standards. Permission was obtained from the database owners to access and use the SNDS database for this study.