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Article History

Received: 8 May 2025

Accepted: 27 November 2025

First Online: 9 December 2025

Declarations

:

: This study was approved by the Internal Review Board at University of Colorado COMIRB 19-2756, with reliance agreements at each clinical site, and University of Western Australia (RA/4/20/6198). The International CDKL5 Clinical Research Network (ICCRN) is registered with ClinicalTrials.gov (NCT0555837). Parents provided informed consent before participation.

: All participants provided consent to participate including that their data would be published but with identifying information.

: Author JD: Consultancy for Marinus, Ultragenyx, Acadia, Avexis, Orion, Takeda, Neurogene, Taysha and Neurocrine. Clinical Trials with Anavex and Newron. Consulting/Advisory Board member for SCN2A Australia. All remuneration has been made to her department. Author JK: Consultancy for Taysha. All remuneration has been made to her department. Author HL: Consultancy for Marinus, Acadia, Avexis, Orion, Neurogene and Taysha. Clinical Trials with Anavex and Newron. All remuneration has been made to her department. Author EM: Site PI for industry sponsored trials of SK Life, Takeda, Epygenix, Ionis, UCB pharma (Zogenix), and Acadia Pharmaceuticals. Grant funding from NIH, State of Pennsylvania and IRSF, RSRT, and LouLou foundation. Consulting/Advisory Board member for Acadia Pharmacueticals, Stoke, and Taysha therapeutics. Author TB: Research funding from GRIN2B Foundation, the International Foundation for CDKL5 Research, Loulou Foundation, the National Institutes of Health, and Simons Foundation. Consultancy for Alcyone, AveXis, GRIN Therapeutics, GW Pharmaceuticals, the International Rett Syndrome Foundation, Marinus Pharmaceuticals, Neurogene, Ovid Therapeutics, and Takeda Pharmaceutical Company Limited. Clinical trials with Acadia Pharmaceuticals Inc., GW Pharmaceuticals, Ionis, Marinus Pharmaceuticals, Neurogene, Ovid Therapeutics, and Rett Syndrome Research Trust. All remuneration has been made to his department. Author SD: Consulted for Biomarin, Neurogene, Marinus, Tysha, Ultragenyx, UCB, Capsida, Encoded, Longboard, Mahzi therapeutics, and Ovid Therapeutics. Funding from NIH, Project 8P and Mila’s Miracle Foundation. Advisory board member for non-profit foundations Rare X, SLC6A1 Connect, Project 8P, Ring14 USA, FamilieSCN2A and N of 1 Collaborative. None of the other authors have any declarations of interest.