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Authors
Hendrickx, Niels
Mentré, France
Hamdan, Alzahra
Karlsson, Mats O.
Hooker, Andrew C.
Traschütz, Andreas
Gagnon, Cynthia
Schüle, Rebecca
Synofzik, Matthis
Comets, Emmanuelle
,
Chen, Xiaomei
Heussen, Nicole Maria
Hilgers, Ralf-Dieter
Klockgether, Thomas
Ryeznik, Yevgen
Sverdlov, Oleksandr
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Funding
Funding for this research was provided by:
EJPRD (825575, 825575, 825575)
TreatHSP network (01GM2209A)
ERDERA (101156595)
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Article History
Received: 12 February 2025
Accepted: 27 June 2025
First Online: 30 July 2025
Declarations
:
: For this study, we used a previously published model to simulate data, so no personal data was collected and/or processed.
: Not applicable.
: Dr. Synofzik has received consultancy honoraria from Ionis, UCB, Prevail, Orphazyme, Servier, Reata, Biogen, GenOrph, AviadoBio, Biohaven, Zevra, Solaxa, and Lilly, all unrelated to the present manuscript. Dr Emmanuelle Comets has received consultancy honoraria from Sanofi, unrelated to the present manuscript. Drs Hooker and Karlsson have received consultancy fees from and own stock in Pharmetheus, all unrelated to this manuscript. Dr. Mentré has received consultancy fees from Pharmetheus and Ipsen, all unrelated to this manuscript. All other authors declared no competing interests in this work.
