Wong, Kingsley
Junaid, Mohammed
Alexander, Solomon
Olson, Heather E.
Pestana-Knight, Elia M.
Rajaraman, Rajsekar R.
Downs, Jenny
Leonard, Helen http://orcid.org/0000-0001-6405-5834
Funding for this research was provided by:
Takeda Pharmaceuticals U.S.A.
Zogenix
Ovid Therapeutics
Marinus Pharmaceuticals
UCB US
International Foundation for CDKL5 Research
Neurogene
Ultragenyx Pharmaceutical
ACADIA Pharmaceuticals
Novartis Gene Therapies
Orion Corporation
Taysha Gene Therapies
FOXG1 Research Foundation
Anavex Life Science
Newron Pharmaceuticals
Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania (CDKL5-19-D-101-3)
University of Western Australia
Article History
Accepted: 19 June 2024
First Online: 26 July 2024
Declarations
:
: Open Access funding enabled and organized by CAUL and its Member Institutions. The International CDKL5 Disorder Database was funded by the International Foundation for CDKL5 Research, along with the Orphan Disease Centre at the University of Pennsylvania (CDKL5-19-D-101-3) for analytical support for this study.
: Heather E. Olson received consulting fees from Takeda Pharmaceuticals and Zogenix regarding the clinical trial design, Ovid Therapeutics regarding clinical trial results and Marinus Pharmaceuticals regarding CDKL5 deficiency disorder, and has done consulting for the FOXG1 Research Foundation. She is the site Principal Investigator for a trial with UCB Pharmaceuticals. Elia M. Pestana-Knight was a speaker and scientific advisory board member for Marinus Pharmaceuticals, for which she has received compensation. Rajsekar R. Rajaraman was a speaker/consultant for Marinus Pharmaceuticals. He was also a consultant for UCB Pharmaceuticals and received funding from the International Foundation for CDKL5 Research. Jenny Downs provided consultancy for Marinus Pharmaceuticals, Neurogene, Ultragenyx Pharmaceutical, Acadia Pharmaceuticals, Novartis Gene Therapies, Orion Corporation and Taysha Gene Therapies; and for clinical trials with Anavex Life Science and Newron Pharmaceuticals. All consultancies are unrelated to this work, and all remuneration has been made to her department. Helen Leonard provided consultancy for Marinus Pharmaceuticals, Acadia Pharmaceuticals, Novartis Gene Therapies and Orion Corporation; and for clinical trials with Anavex Life Science and Newron Pharmaceuticals. All consultancies are unrelated to this work, and all remuneration has been made to her department. Kingsley Wong, Mohammed Junaid and Solomon Alexander have no conflicts of interest that are directly relevant to the content of this article.
: The Human Research Ethics Committee of the University of Western Australia provided approval for this study.
: Written informed consent was obtained from the caregivers of individuals who participated in the study.
: Not applicable.
: All authors contributed to the study’s conception and design. M.J., K.W. and S.A. performed material preparation, data collection and analysis. H.L., M.J. and K.W. wrote the first draft of the manuscript, and all authors commented on previous versions. All authors read and approved the final manuscript.
: The dataset analysed during the current study are not publicly available but may be available from the corresponding author on reasonable request following an application to and with approval from the local ethics committee.
: Not applicable.