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Authors
Morison, Lottie D. https://orcid.org/0000-0003-2822-6522
Vogel, Adam P. https://orcid.org/0000-0002-3505-2631
Christodoulou, John https://orcid.org/0000-0002-8431-0641
Gold, Wendy A.
Verden, Dylan
Chung, Wendy K. https://orcid.org/0000-0003-3438-5685
Braden, Ruth
Bredebusch, Joanna
Kaur, Simranpreet
Scheffer, Ingrid E.
Morgan, Angela T. https://orcid.org/0000-0003-1147-7405
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Article History
Received: 2 March 2025
Revised: 23 April 2025
Accepted: 30 April 2025
First Online: 16 May 2025
Competing interests
: DV is the Research Engagement Director at KIF1A.org. APV is the Chief Science Officer and ATM is the director of paediatric/neurodevelopment communication at Redenlab Pty Ltd. JC is a member of the Anavex Drug Safety and Monitoring Board for a Rett syndrome drug trial and is not in receipt of any remuneration for this involvement. JC is an expert advisor for Taysha Gene Therapies which is running a Rett syndrome gene therapy trial and is receiving remuneration for this involvement. IES is a consultant for Biohaven Pharmaceuticals, Care Beyond Diagnosis, Cerecin Inc, Eisai, Epilepsy Consortium, Longboard Pharmaceuticals, UCB, Zynerba Pharmaceuticals. IES has received payment honoraria and travel support from Biocodex, BioMarin, Chiesi, Eisai, GlaxoSmithKline, Liva Nova, Nutricia, Stoke Therapeutics, UCB, Zuellig Pharma. IES is on scientific advisory boards for Bellberry Ltd, BioMarin, Chiesi, Eisai, Encoded Therapeutics, Garvan Institute of Medical Research, Knopp Biosciences, Longboard Pharmaceuticals, UCB, Takeda Pharmaceuticals. IES is a director at Bellberry Ltd, Australian Academy of Health and Medical Sciences, Australian Council of Learned Academies Ltd. IES is a trial investigator for Anavex Life Sciences, Cerebral Therapeutics, Cerecin Inc, Cereval Therapeutics, Eisai, Encoded Therapeutics, EpiMinder Inc, ES-Therapeutics, GW Pharmaceuticals, Marinus Pharmaceuticals, Neuren Pharmaceuticals, Neurocrine BioSciences, Ovid Therapeutics, Takeda Pharmaceuticals, UCB, Ultragenyx, Xenon Pharmaceuticals, Zogenix, Zynerba Pharmaceuticals. IES has a patent issued for # Molecular diagnostic/theranostic target for benign familial infantile epilepsy (BFIE) [PRRT2] 2011904493 & 2012900190 and PCT/AU2012/001321 (TECH ID:2012-009) # SCN1A testing held by Bionomics Inc and a patent pending for WO61/010176 (filed: 2008): Therapeutic Compound. All other authors have no competing interests to declare.
: Ethics approval was obtained from the Royal Children’s Hospital Human Research Ethics Committee (HREC 37353A), Columbia University and Boston Children’s Hospital institutional review boards. All participants/caregivers provided informed consent.
