May, Damian
Kponee-Shovein, Kalé
Neul, Jeffrey L.
Percy, Alan K.
Mahendran, Malena
Downes, Nathaniel
Chen, Grace
Watson, Talissa
Pichard, Dominique C.
Kennedy, Melissa
Lefebvre, Patrick
Funding for this research was provided by:
ACADIA Pharmaceuticals (ACADIA Pharmaceuticals)
Article History
Received: 5 February 2024
Accepted: 2 July 2024
First Online: 26 July 2024
Declarations
:
: Data were de-identified and certified as fully compliant with US patient confidentiality requirements outlined in the Health Insurance Portability and Accountability Act (HIPAA). Since this study relied exclusively on de-identified patient records and did not involve the collection, use, or dissemination of individually identifiable data, institutional review board approval was not required as per HIPAA. All methods were carried out in accordance with relevant guidelines and regulations in the Declaration of Helsinki. The data that support the findings of this study are not publicly available online; administrative permissions to access the data were given by the International Rett Syndrome Foundation under license.
: Not applicable.
: DM is an employee of Acadia Pharmaceuticals Inc. DCP and MK are employees of the International Rett Syndrome Foundation. KKS, MM, ND, GC, TW, and PL are employees of Analysis Group, Inc., a consultancy that received funding from Acadia Pharmaceuticals, Inc. to conduct this study. AKP is co-editor of <i>Translational Science of Rare Diseases</i>, received research funding from the National Institutes of Health, and is a consultant for Acadia Pharmaceuticals Inc., Anavex Life Sciences Corp., AveXis, and GW Pharmaceuticals, as well as advisor to the International Rett Syndrome Foundation. JLN 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 Inc., 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 Peer Review 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.