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Article History

Received: 18 October 2024

Accepted: 6 January 2025

First Online: 24 January 2025

Declarations

:

: This study was reviewed and approved by the Food and Drug Administration, by the institutional review boards at each participating site, and was conducted in accordance with the principles of the Declaration of Helsinki and Good Clinical Practice. Appropriate written informed consent for study participation was obtained prior to completing any study procedures.

: Not applicable.

: KC has no competing interests. EBK has no competing interests. DNL has acted as a consultant for Acadia Pharmaceuticals, Neurogene, and Taysha Gene Therapies. He has been a Site PI for industry sponsored trials from Acadia Pharmaceuticals, GW Pharmaceuticals, Anavex Life Sciences, and Neurogene. He has received research support from RSRT. TAB has 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, Takeda Pharmaceutical Company Limited and Taysha; clinical trials with Acadia Pharmaceuticals Inc., GW Pharmaceuticals, Marinus Pharmaceuticals, Neurogene, Ovid Therapeutics, and Rett Syndrome Research Trust; all remuneration has been made to his department. CF has been a site investigator for industry sponsored clinical trials from Zogenix pharmaceuticals, Acadia pharmaceuticals, GW pharmaceuticals, Neurogene, and Taysha. AP was a site investigator for Acadia Pharmaceuticals and is a consultant for Acadia, Neurogene, and Taysha Gene Therapies. BS has received research funding from the NIH, IRSF, and the Blue Bird Circle; he has provided consultancy for IONIS pharmaceuticals, Neurogene, and Taysha; clinical trials with Acadia Pharmaceuticals Inc., Marinus Pharmaceuticals, Neurogene, and the Rett Syndrome Research Trust. EDM has acted as a consultant for Stoke therapeutics, Acadia Pharmaceuticals, and Novartis Pharmaceuticals. He has been a Site PI for industry sponsored trials for Stoke Theraputics, Acadia Pharmaceuticals, GW Pharmaceuticals, Zogenix Pharmaceuticals, Marinus Pharmaceuticals, Takeda Pharmaceuticals and Epygenic Pharmaceuticals. He has received research support from the NIH, Penn Orphan Disease center, RettSyndrome.org, RSRT, and International CDKL5 Research Foundation. DM has no competing interests. RLC has no competing interests. JLN has acted as a consultant for Acadia Pharmaceuticals, Alycone Pharmaceuticals, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, IONIS Pharmaceuticals, Neurogene, Newron Pharmaceuticals, Ovid Therapeutics, Taysha Gene Therapies, and Ultragenyx. He has been a site PI for industry sponsored clinical research for Acadia Pharmaceuticals, GW Pharmaceuticals, IONIS Pharmaceuticals, Newron Pharmaceuticals. He has received research support from the National Institutes of Health, International Rett Syndrome Foundation, and RSRT. JvH has no competing interests.