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Article History

Received: 29 November 2023

Accepted: 3 May 2024

First Online: 13 June 2024

Declarations

:

: Institutional review board approval was obtained at each site before study initiation. Written consent was obtained from each guardian (or adult participants in the case of individuals who were capable to provide their own consent).

: Not applicable.

: AJK, JK, AD, EC, ES, D. Harvey and KR: no relevant disclosures; EBK has received funding from the following, all of which are directed to Rush University Medical Center in support of rare disease programs, and she receives no personal funds and has no relevant financial interest in any of the commercial entities listed: Acadia, Alcobra, Anavex, Biogen, BioMarin, Cydan, Fulcrum, GeneTx, GW, Ionis, Lumos, Marinus, Neuren, Neurotrope, Novartis, Orphazyme, Ovid, Roche, Seaside Therapeutics, Tetra, Ultragenyx, Yamo, and Zynerba to consult on trial design and development strategies and/or to conduct clinical studies in FXS or other NNDs or neurodegenerative disorders; Vtesse/Sucampo/Mallinckrodt Pharmaceuticals to conduct clinical trials in Nieman Pick; and Asuragen Inc to develop testing standards for <i>FMR1</i> testing; D. Hessl has received funding from the following, all of which are directed to the UC Davis, in support of fragile X treatment programs, and he receives no personal funds and has no relevant financial interest in any of the commercial entities listed: Autifony, Ovid, Tetra/Shionogi, Healx, and Zynerba pharmaceutical companies to consult on outcome measures and clinical trial design. D. Hessl and EBK are members of the Clinical Trials Committee of the National Fragile X Foundation.