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Article History

Received: 21 April 2021

Accepted: 28 February 2022

First Online: 14 May 2022

Declarations

:

: Parental consent for study conduct and publication of results was obtained prior to entry into the study for all participants. Each participating institution retained institutional review board approval for the implementation of this study protocol and consent form (ClinicalTrials.gov identifiers: NCT00299312 and NCT02738281).

: Not applicable.

: Caroline B. Buchanan received funding from Rettsyndrome.org (Mentored Clinical Fellowship under U54 HD 061222 grant) and has done clinical trials with Acadia, Anavex, GW Pharmaceuticals, and Zynerba. Timothy A. Benke received funding from the NIH (U54 HD061222, R21 NS101288, R01 NS081248), International Foundation for CDKL5 Research, Loulou Foundation, and Children’s Hospital Colorado Foundation Ponzio Family Chair in Neurology Research. He is also a consultant for AveXis, Ovid, GW Pharmaceuticals, International Rett Syndrome Foundation, Takeda, and Marinus and has done clinical trials with Acadia, Ovid, GW Pharmaceuticals, Marinus, and Rett Syndrome Research Trust; all remuneration has been made to his department. Daniel G Glaze received funding from the NIH, Rettsyndrome.org, and the Blue Bird Circle. He has done clinical trials with Acadia, Neuren, and GW Pharmaceuticals. Richard H. Haas received funding from the NIH 5U54HD061222 grant and has done clinical trials sponsored by Acadia, GW Pharmaceuticals, and Newron Pharmaceuticals. Jane B. Lane received funding from the NIH and is a consultant for International Rett Syndrome Foundation and GW Pharmaceuticals. David N. Lieberman has done clinical trials with Anavex, Acadia, GW Pharmaceuticals, and Rett Syndrome Research Trust. Eric D. Marsh received funding from Rett Syndrome Research Trust, NIH, State of Pennsylvania, Penn Orphan Disease Center, International Rett Syndrome Foundation, LouLou Foundation, and Eagles Autism Foundation. He is a consultant for Cilpa Therapeutics and Stoke Therapeutics. He has done clinical trials with Zogenix, GW Pharmaceuticals, Acadia, Marinus, Stoke Therapeutics, and Rett Syndrome Research Trust. Jeffrey L. Neul received funding from the NIH and is a consultant for AveXis, Biohaven, Eloxx Pharmaceuticals, Ovid, Takeda, and Teva Pharmaceuticals. He has done clinical trials with Acadia and Newron Pharmaceuticals. Sarika U. Peters received funding from NIH, Rettsyndrome.org and is a consultant for Acadia Pharmaceuticals. Steve A. Skinner has done clinical trials with Acadia, Anavex, and GW Pharmaceuticals. Shannon M. Standridge is a speaker for Greenwich Biosciences and Scientific Advisory Board of Acadia. Walter E. Kaufmann received funding from the NIH and CDC, and is a consultant for Anavex, AveXis, Acadia, EryDel, Newron, GW Pharmaceuticals, Marinus, Biohaven, Zynerba, Ovid Therapeutics, and Stalicla. Currently, he is Chief Medical Officer of Anavex Life Sciences Corp. Alan K. Percy received funding from the NIH and is a consultant for Anavex, AveXis, Acadia, and GW Pharmaceuticals. He has done clinical trials with Anavex, Acadia, GW Pharmaceuticals, and Rett Syndrome Research Trust. The rest of the authors, Jennifer L. Stallworth, Aubin E. Joy, Rebekah E. Dixon, Alexandra E. Scott, Arthur A. Beisang, Peter T. Heydemann, Mary D. Jones, and Robin C. Ryther, declare that they have no competing interests.