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Article History

Received: 12 September 2024

Accepted: 2 December 2024

First Online: 21 December 2024

Declarations

:

: Workshop involving literature review, ethics approval not necessary for this study.

: Not necessary for this study. Workshop participants are the authors for this manuscript.

: JD: Consultancy for Marinus, Ultragenyx, Acadia, Avexis, Orion, Neurogene, Taysha and Takeda; Clinical Trials with Anavex and Newron. All consultancies are unrelated to this work and all remuneration has been made to her department.DCP: None.WEK: Past Chief Scientific Officer of Anavex Life Sciences Corp. He received funding from the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC), and he has been a consultant for Anavex, Acadia, EryDel/Quince, Neuren, Newron, GW Pharmaceuticals, Marinus, Biohaven, Zynerba, Ovid Therapeutics, Stalicla, Unravel Biosciences and Tetra Therapeutics. He has done clinical trials with Ipsen and Neuren.JPH: Employee at AMO Pharma, based in Wornesh, United Kingdom.MR: None.GT: None.EM: The PI for Stoke therapeutics, Zogenix Pharmaceuticals, Acadia Pharmaceuticals, Marinus Pharmaceuticals, Takeda Pharmaceuticals, Epygenix Pharmaceuticals. He has received research support from NIH, Penn Orphan Disease center, International Rett syndrome foundation, International CDKL5 Research Foundation, Curaleaf Inc., is a consultant for Acadia Pharmaceuticals, and has prepared an educational programme for Medscape.HL: Consultancy for Marinus, Acadia, Avexis and Orion; Clinical Trials with Anavex and Newron; All remuneration has been made to her department.KM: None.ACD: Work completed while an employee and equity holder of Shape Therapeutics, Inc. Current employee and equity holder of Ensoma, which had no oversight or funding of this work.NG: None.AA: Research funding from Acadia Pharmaceuticals, Zogenix Inc. Consulting for Sanofi, Acadia.BB: Currently research funding from the NIH (NICHD) and Acadia Pharmaceuticals.SP: Dr. Peters has received research funding from the MECP2 Duplication Foundation, the ActiGraph Corporation, and the National Institutes of Health. She is a consultant for Ionis Pharmaceuticals and Acadia Pharmaceuticals.CB: Consultant for Acadia Pharmaceuticals.FS: None.AJ: None.JY: Employee of Acadia Pharmaceuticals.BS: Received research funding from the International Foundation for CDKL5 Research, Loulou Foundation, the National Institutes of Health, International Rett Syndrome Foundation, the Rett Syndrome Research Trust, and the Grace Science Foundation; consultancy for the IONIS pharmaceuticals, Neurogene, and Taysha; clinical trials with Acadia Pharmaceuticals Inc., Marinus Pharmaceuticals, Neurogene, and the Rett Syndrome Research Trust. All remuneration has been paid to Baylor College of Medicine.PS: Lead of the CIPP Rett Centre, UK, which is partially funded by Reverse Rett, UK. P.S. was a previous Principal Investigator (PI) in the Sarizotan (Protocol Number Sarizotan/001/II/2015; ClinicalTrials.gov Identifier: NCT02790034) and the Anavex Life Sciences Corp. (Protocol Number: ANAVEX2-73-RS-002) clinical trial on Rett Syndrome (RTT). P.S. is currently the PI in the Anavex Life Sciences Corp. (Protocol Number: ANAVEX2-73-RS-003) clinical trial. P.S. is the co-inventor of the HealthTrackerTM and is the Chief Executive Officer and shareholder in HealthTrackerTM.JLN: Received research funding from the National Institutes of Health, the International Rett Syndrome Foundation, and Rett Syndrome Research Trust; served as investigator for clinical trials conducted by Acadia Pharmaceuticals, GW Pharmaceuticals, Neuren Pharmaceuticals, and Newron; received personal consultancy for Acadia Pharmaceuticals Inc, Analysis Group, Anavex, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Ionis Pharmaceuticals, Myrtelle, Neurogene, Newron Pharmaceuticals, Signant Health, and Taysha Gene Therapies, and the preparation of CME activities for the France Foundation, MedEdicus, Medscape, PeerView Institute, Medscape, TotalCME; served on the scientific advisory board of Alcyone Lifesciences; was a scientific co-founder of LizarBio Therapeutics; and a member of a data safety monitoring board for clinical trials conducted by Ovid Therapeutics and Ultragenix.TAB: Received 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, Marinus Pharmaceuticals, Ovid Therapeutics, and Rett Syndrome Research Trust; all remuneration has been made to his department.