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Article History

Received: 5 December 2023

Accepted: 6 August 2024

First Online: 13 August 2024

Declarations

:

: This study was conducted in accordance with the ISPOR Code of Ethics 2017, Good Epidemiology Practices and with the ethical principles laid down in the Declaration of Helsinki. Compliance with these regulatory standards and guidance provided assurance that the rights, safety and wellbeing of subjects participating in this study are protected. Approval to conduct the study was obtained from ethics committees to comply with local laws and regulations in the United States, Europe and Australia; written or electronic informed consent was obtained for all participants.

: Not applicable.

: WEK is currently the 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, AveXis, Acadia, EryDel, Neuren Pharmaceuticals, Newron, GW Pharmaceuticals, Marinus, Biohaven, Zynerba, Ovid Therapeutics, and Stalicla. He has done clinical trials with Neuren. AKP received funding from the NIH and is a consultant for Anavex, Taysha Gene Therapies, Neurogene, and Acadia. He is a member of the DSMB for Taysha Gene Therapies and has done clinical trials with Anavex, Acadia, GW Pharmaceuticals, and the Rett Syndrome Research Trust (RSRT). JLN received funding from the NIH, International Rett Syndrome Foundation (IRSF) and RSRT. He is a consultant for Acadia, Analysis Group, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Myrtelle, Neurogene, Newron, Signant Health, Taysha Gene Therapies, and for the preparation of CME activities for PeerView Institute MedEdicus and Medscape; he serves on the scientific advisory board of Alcyone Lifesciences and is a scientific cofounder of LizarBio Therapeutics; and he was also a member of a data safety monitoring board for clinical trials conducted by Ovid Therapeutics. He has done clinical trials with Acadia and Newron. JD has consulted for Marinus, Newron, Orion, Taysha, Neurogene, Acadia and AveXis. She has done clinical trials with Newron and Anavex. HL has consulted for Acadia, Avexis, Anavex, GW Pharmaceuticals, Newron, Neurogene, Marinus, and Ovid Therapeutics. She has received funding from the NIH, International Foundation for CDKL5 Research and the Loulou Foundation. HMO, KVB, ERH, and CUM are Anavex employees. VV-F is a consultant for Anavex. The rest of the authors, PN, GDS, TEB, GST, LMGC, OM, and CB, declare that they have no competing interests.