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Authors
Aslanzadeh, Vahid
Brierley, Gemma V.
Kumar, Rupa
Çubuk, Hasan
Vigouroux, Corinne https://orcid.org/0000-0002-8181-4721
Matreyek, Kenneth A. https://orcid.org/0000-0001-9149-551X
Kudla, Grzegorz https://orcid.org/0000-0002-7924-2744
Semple, Robert K. https://orcid.org/0000-0001-6539-3069
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Funding
Funding for this research was provided by:
Wellcome Trust (210752, 207507)
British Heart Foundation (RE/18/5/34216)
Diabetes UK (22/0006407, 22/0006407)
French Ministry of Health and Assistance-Publique Hôpitaux de Paris PRISIS rare disease reference centre European Reference Network on Rare Endocrine Conditions
RCUK | Medical Research Council (MC_UU_00035/8)
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Article History
Received: 8 March 2025
Accepted: 5 September 2025
First Online: 15 October 2025
Competing interests
: R.K.S. has received consulting fees from Novartis, Astra Zeneca, and Alnylam, research contribution in kind from Pfizer, and speaking fees from Novo Nordisk, Eli Lilly, and Amryt. CV serves as investigator in the APL-22 clinical study sponsored by Chiesi Farmaceutici and in the REGN4461-PLD-20100 sponsored by Regeneron Pharmaceuticals, and has served as speaker and received support for attending meetings from Amryt Pharmaceuticals (now Chiesi Farmaceutici) and Sanofi. The remaining authors declare no competing interests.
