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Article History

Received: 10 January 2023

Accepted: 12 May 2023

First Online: 8 June 2023

Competing interests

: J.L.N. has received research funding from the International Rett Syndrome Foundation, the National Institutes of Health and Rett Syndrome Research Trust; and personal consultancy fees from Acadia Pharmaceuticals, Analysis Group, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Myrtelle, Neurogene, Newron Pharmaceuticals, Signant Health and Taysha Gene Therapies and for the preparation of CME activities for Medscape and PeerView Institute; 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. A.K.P. is coeditor of <i>Translational Science of Rare Diseases</i>, received research funding from the National Institutes of Health and is a consultant for Acadia Pharmaceuticals, AveXis, GW Pharmaceuticals and Anavex Life Science as well as an adviser to the International Rett Syndrome Foundation. T.A.B. received research funding from the GRIN2B Foundation, the International Foundation for CDKL5 Research, the Loulou Foundation, the National Institutes of Health and the Simons Foundation; has consultancies for Alcyone, AveXis, GRIN Therapeutics, GW Pharmaceuticals, the International Rett Syndrome Foundation, Marinus Pharmaceuticals, Neurogene, Ovid Therapeutics and Takeda Pharmaceutical; has clinical trials with Acadia Pharmaceuticals, GW Pharmaceuticals, Marinus Pharmaceuticals, Ovid Therapeutics and Rett Syndrome Research Trust; all remuneration has been made to his department. E.M.B.-K. has received funding from Acadia Pharmaceuticals, Alcobra Pharmaceuticals, AMO Pharma, Asuragen, AveXis, Biogen, BioMarin, Cydan Development, EryDel, Fulcrum Therapeutics, GeneTx, GW Pharmaceuticals, Ionis Pharmaceuticals, Jaguar Health, Lumos Pharma, Marinus Pharmaceuticals, Neuren Pharmaceuticals, Neurogene, Neurotrope, Novartis, Orphazyme, Ovid Therapeutics, Retrophin, Roche, Seaside Therapeutics, Taysha Gene Therapies, Tetra Bio-Pharma, Ultragenyx, Yamo Pharmaceuticals, Zynerba Pharmaceuticals and Vtesse–Sucampo–Mallinckrodt Pharmaceuticals to consult on trial design or run clinical or laboratory validation trials in genetic neurodevelopmental or neurodegenerative disorders, all of which is directed to Rush University Medical Center in support of rare disease programs; E.M.B.-K. receives no personal funds, and Rush University Medical Center has no relevant financial interest in any of the commercial entities listed. D.G.G. has received personal compensation and research support from Acadia Pharmaceuticals, Neuren Pharmaceuticals and Newron Pharmaceuticals. E.D.M. has received research support from the National Institute of Neurological Disorders and Stroke and the National Institute of Child Health and Human Development, the Eagles Autism Foundation, Penn Orphan Disease Center, the International Rett Syndrome Foundation, Rett Syndrome Research Trust, the International CDKL5 Research Foundation and the Loulou Foundation. He has been a site principal investigator for trials from Stoke Therapeutics, GW Pharmaceuticals, Zogenix, Acadia Pharmaceuticals and Marinus Pharmaceuticals. He has received personal compensation for consulting from Stoke Therapeutics and Acadia Pharmaceuticals. J.M.Y., K.M.B., S.S. and T.L. are employees of and stockholders in Acadia Pharmaceuticals. S.S. is also a board director and stockholder of Neurogene.