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Authors
Biswas, Susmito https://orcid.org/0000-0003-1548-0572
Casteels, Ingele https://orcid.org/0000-0002-2335-1845
Bremond-Gignac, Dominique https://orcid.org/0000-0002-3563-5482
Hogan, Julien https://orcid.org/0000-0003-4838-9417
Iarossi, Giancarlo https://orcid.org/0000-0002-6302-5542
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Article History
Received: 7 July 2025
Accepted: 11 August 2025
First Online: 31 August 2025
Declarations
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: Susmito Biswas, Julien Hogan and Giancarlo Iarossi have received speaker honoraria from Recordati Rare Diseases. Dominique Bremond-Gignac and Ingele Casteels have nothing to disclose.
: The study was conducted in accordance with the Declaration of Helsinki 1964 and all subsequent amendments, and approved by the appropriate ethics Committees of Belgium, Italy, France and the UK. The ethics boards are: Ethische Commissie Onderzoek UZ/KU Leuven, Campus Gasthuisberg E330, Herestraat 49, 3000 Leuven, Belgium; Bambino Gesù Hospital, Rome, Italy; Agence Nationale de Sécurité du Médicament et des Produits de Santé, France; Health Research Authority (HRA) and Health and Care Research Wales (HCRW). Protocol number CYT-C2-001, date of approval 18/09/19. Written informed consent was obtained from each patient’s parents or legally acceptable representatives prior to any study procedures being performed. As patients were under 2 years of age, they were not required to provide assent for their participation in the study.
