Brügelmann, Malina
Müller, Sophia
Bohlen, Alina V.
Hohenfellner, Katharina
Büscher, Anja
Kemper, Markus J.
Fröde, Kerstin
Kanzelmeyer, Nele
Oh, Jun
Billing, Heiko
Gellermann, Jutta
Müller, Dominik
Weber, Lutz T.
Acham-Roschitz, Birgit
Arbeiter, Klaus
Tönshoff, Burkhard
Hagenberg, Martina
Žebec, Mislav S.
Haffner, Dieter
Zivicnjak, Miroslav http://orcid.org/0000-0001-9302-712X
Funding for this research was provided by:
Horizon Pharmaceuticals
Article History
Received: 25 January 2024
Revised: 7 May 2024
Accepted: 21 May 2024
First Online: 8 June 2024
Declarations
:
: The study received appropriate ethics committee approval from the Institutional Review Board at each site and was performed in accordance with the Declaration of Helsinki.
: Written informed consent was obtained from all parents/guardians, with consent or assent from patients where age appropriate.
: Dieter Haffner received speaker fees and research grants from Horizon and Chiesi. Jun Oh received speaker fees from Horizon and Chiesi. Burkhard Tönshoff participated in advisory boards for Chiesi. Lutz T. Weber received speaker fees from Chiesi. Katharina Hohenfellner was supported by the Cystinosis Foundation Germany. All other authors declare that they have no conflict of interest.