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Authors
Seefried, Lothar https://orcid.org/0000-0003-1154-3388
Rak, Dominik https://orcid.org/0000-0003-3118-6525
Genest, Franca https://orcid.org/0000-0001-6233-4805
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Funding
Funding for this research was provided by:
Alexion Pharmaceuticals
Julius-Maximilians-Universität Würzburg
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Article History
Received: 25 November 2024
Accepted: 9 April 2025
First Online: 16 April 2025
Declarations
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: L. Seefried is a clinical study investigator and has received consultancy fees and institutional research funding and/or grant support from Alexion, AstraZeneca Rare Disease; Amgen; AM-Pharma; BioMarin; Chiesi; Haleon/GSK; Inozyme; Ipsen; Kyowa Kirin; medi; Novartis; STADApharm; Theramex; UCB; and Ultragenyx. D. Rak has no conflict of interest to declare. F. Genest is a clinical study investigator and has received speaker honoraria from Alexion, AstraZeneca Rare Disease.
