Mekahli, Djalila http://orcid.org/0000-0003-0954-6088
Guay-Woodford, Lisa M. http://orcid.org/0000-0002-6187-3927
Cadnapaphornchai, Melissa A. http://orcid.org/0000-0002-0460-2477
Goldstein, Stuart L. http://orcid.org/0000-0003-3711-8785
Dandurand, Ann
Jiang, Huan
Jadhav, Pravin http://orcid.org/0000-0001-5498-2124
Debuque, Laurie
Funding for this research was provided by:
Otsuka Pharmaceutical Development and Commercialization
Article History
Received: 11 July 2023
Revised: 6 November 2023
Accepted: 8 November 2023
First Online: 13 December 2023
Declarations
:
: As reported previously (Schaefer F et al., <i>Eur J Pediatr</i> 2019;178:1013–1021), the clinical trial from which the data analyzed here were obtained was conducted in conformity with FDA regulations, International Conference on Harmonization Good Clinical Practice Guideline (E6), international ethical principles derived from the Declaration of Helsinki, Council for International Organizations of Medical Science guidelines, and applicable local laws and regulations.
: As reported previously for the original clinical trial from which the data analyzed here were obtained (Schaefer F et al., <i>Eur J Pediatr</i> 2019;178:1013–1021), written informed consent was obtained from a parent/guardian or legally acceptable representative, as applicable for local laws, at screening, prior to the initiation of any protocol-required procedures. In addition, the subject must provide age-appropriate informed assent at screening and must have been able to understand that he or she could withdraw from the trial at any time.
: Dr. Mekahli, as a representative of UZ Leuven and KU Leuven, reports research grants from Otsuka Pharmaceuticals, and serves on advisory boards for Otsuka Pharmaceuticals, Sanofi Genzyme, and Reata, all paid to her institutions UZ Leuven and KU Leuven. She is supported by the Research Foundation Flanders (FWO) (G0C8920N and G060623N) and clinical senior research grant for D. Mekahli (1804123N).Dr. Guay-Woodford serves a consultant for and receives honoraria from Otsuka and Natera, Inc.Dr. Cadnapaphornchai serves on a pediatric advisory board of Otsuka Pharmaceuticals Inc.Dr. Goldstein has received grant funding from and served as a consultant for Otsuka.Dr. Dandurand was formerly employed by Otsuka.Dr. Jiang is an employee of Otsuka.Dr. Jadhav was formerly employed by Otsuka and serves as a consultant for Otsuka.Ms. Debuque is an employee of Otsuka.