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Article History

Received: 8 February 2023

Accepted: 20 September 2023

First Online: 13 October 2023

Declarations

:

: This study was approved by Institutional Review Board (IRB) either under a single-IRB agreement provided by the University of Alabama at Birmingham or the respective institutions. Written informed consent was obtained for each participant according to the Declaration of Helsinki. A Certificate of Confidentiality was provided by the National Institute of Child Health and Development (NICHD). This non-interventional clinical trial protocol was registered with Clinical Trials.Gov (NCT02738281).

: Not applicable.

: JLN received research funding from the National Institutes of Health, the International Rett Syndrome Foundation, and Rett Syndrome Research Trust; clinical trials with Acadia Pharmaceuticals Inc., GW Pharmaceuticals; personal consultancy for Acadia Pharmaceuticals Inc., Analysis Group, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Myrtelle, Neurogene, Newron Pharmaceuticals, Signant Health, Taysha Gene Therapies, and the preparation of CME activities for PeerView Institute, MedEdicus, and Medscape; serves on the scientific advisory board of Alcyone Lifesciences; is a scientific cofounder of LizarBio Therapeutics; and was a member of a data safety monitoring board for clinical trials conducted by Ovid Therapeutics.TAB received research funding from GRIN2B Foundation, International Rett Syndrome 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, Marinus Pharmaceuticals, Ovid Therapeutics, and Rett Syndrome Research Trust; all remuneration has been made to his department.EDM received research support from the National Institutes of Health, Penn Orphan Disease Center, the International Rett Syndrome Foundation, Rett Syndrome Research Trust, International CDKL5 Research Foundation, and the Loulou Foundation. He has been a site principal investigator for trials from Stoke Therapeutics, Zogenix, Acadia Pharmaceuticals Inc., Takeda Pharmaceuticals, Epygenix Pharmaceuticals, and Marinus Pharmaceuticals. He has received personal compensation for consulting from Acadia Pharmaceuticals Inc. and the preparation of CME activities for Medscape.BS has been a site investigator for clinical trials with Acadia, Marinus, and Newron; consultancy for Neurogene and Taysha; all remuneration has been paid to his department.LS declares no competing interests.CF has been a site investigator for clinical trials with Acadia.SUP received research funding from the National Institutes of Health, the MECP2 Duplication Foundation, and the ActiGraph Corporation.AKP received research funding from the National Institutes of Health, International Rett Syndrome Foundation, Rett Syndrome Research Trust; clinical trials with Acadia Pharmaceuticals Inc. and Anavex Life Sciences Corp.; and personal consultancy for Acadia Pharmaceuticals Inc. and Anavex Life Sciences Corp.