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Authors
Yap, Sufin https://orcid.org/0000-0002-4417-4876
Gasperini, Serena
Matsumoto, Shirou
Feillet, François
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Article History
Received: 25 June 2024
Accepted: 19 November 2024
First Online: 18 December 2024
Declarations
:
: Not applicable.
: Not applicable.
: SY has received honoraria from Recordati Rare Diseases, Moderna, and Travere Therapeutics, and has acted as clinical advisor to the National Institute for Health and Care Excellence (NICE), UK. SG has received honoraria for participation at conferences and consultation fees from Recordati Rare Diseases, Sanofi, Immedica, Amicus, and Chiesi. FF has received honoraria from BioMarin, Sanofi, Merck Serono, Nutricia International/Danone, Vitaflo, Alexion, Immedica, Travere Therapeutics, and Recordati Rare Diseases. SM was supported by the Japan Society for the Promotion of Science (JSPS) Grants-in-Aid for Scientific Research (KAKENHI) grant numbers JP23K07335, JP22K07948, JP17bk0104008h0005, and JP17bk0104015h0005.
