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Authors
Farrugia, Agnès
Bahey, Ahmed
Tarawah, Ahmad
Al Yamani, Arwa
Abou Selwan, Carla https://orcid.org/0000-0002-8140-8694
Mohty, Dania
Wolfs, Denis
Dan, Dorica
Malhan, Hafiz Mosa Ali
De Graaf, Johan
Živković, Stefan
Wali, Yasser
Ouraybi, Youmna
Al Kadhem, Zakareya
Tawfiq, Nafisa
Davelaar, Lee
Abdelbaky, Noha Mohamed Mahmoud
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Article History
Received: 20 May 2025
Accepted: 15 June 2026
First Online: 31 July 2026
Declarations
Ethics approval and consent to participate: Not applicable.
Consent for publication: Not applicable.
Competing interests: All authors have received honoraria from Pfizer Inc. for participating in the Rare Advocacy Council.
