Article History
Accepted: 27 June 2024
First Online: 9 September 2024
Declarations
:
: No funding supported production of this manuscript.
: A.K.P. has a consulting relationship with Acadia Pharmaceuticals, Anavex, Ionis, Neurogene, and Taysha, has previously served on the DSMB for Taysha, has previously had a contract researcher relationship with Acadia Pharmaceuticals, and has participated in the preparation of CME activities for the France Foundation, MedEdicus, Medscape, PeerView Institute, TotalCME. A.A. has research support from Anavex, Acadia Pharmaceuticals, and UCB. J.L.N. has received research funding from the National Institutes of Health, the International Rett Syndrome Foundation, and Rett Syndrome Research Trust; served as investigator for clinical trials conducted by Acadia Pharmaceuticals, GW Pharmaceuticals, Neuren Pharmaceuticals, and Newron; received personal consultancy for Acadia Pharmaceuticals Inc, Analysis Group, Anavex, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Ionis Pharmaceuticals, Myrtelle, Neurogene, Newron Pharmaceuticals, Signant Health, and Taysha Gene Therapies, and the preparation of CME activities for the France Foundation, MedEdicus, Medscape, PeerView Institute, TotalCME; served on the scientific advisory board of Alcyone Lifesciences; was a scientific cofounder of LizarBio Therapeutics; and a member of a data safety monitoring board for clinical trials conducted by Ovid Therapeutics and Ultragenix.
: This review required no ethics approval. The Natural History Study (NHS) had ethics approval from the relevant institutions during its active period from 2003–2021.
: All individuals enrolled in the NHS were provided informed consent by their parent(s) or guardian and where possible gave assent to be included. Similarly, consent was provided to participate in therapeutic trials.
: Not applicable.
: Data from the US NHS is now managed by the International Rett Syndrome Foundation (IRSF). Access to this information may be obtained by contacting IRSF at .
: Not applicable.
: Alan Percy developed the review and provided the initial draft, performed the literature or on-line searches. Amitha Ananth and Jeffrey Neul critically reviewed and commented on the draft contributed to and approved the final document. All three authors had roles in analysis of the NHS data. All three authors participated in one or more of the clinical trials.