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Article History

Received: 6 April 2021

Accepted: 14 October 2021

First Online: 5 November 2021

Declarations

:

: The Boston Children’s Hospital Institutional Review Board (IRB) approved the study, serving as the central IRB for all the participating study sites. Caregivers and legal guardians of the participants provided informed consent.

: Not applicable.

: SS has received consulting fees from GLG, Guidepoint (which connected to a client, Fortress Biotech), Novartis, ExpertConnect. KK is currently employed by Alexion Pharmaceuticals, Inc. but completed this work while at Boston Children’s Hospital. AK receives research support from AMO Pharma and consults to Ovid Therapeutics, Acadia, and sema4. EBK has received funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren, Cydan, Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Ovid, Retrophin, AMO, Yamo, Acadia, Avexis, Ionis, Ultragenyx, Lumos, GeneTx, and Vtesse/Sucampo/Mallinkcrodt Pharmaceuticals to consult on trial design or development strategies and/or conduct clinical trials in FXS or other NDDs or neurodegenerative disorders, and from Asuragen Inc to develop testing standards for FMR1 testing. All funding to EBK is directed to Rush University Medical Center to support rare disease programs. EBK receives no personal funds. MS reports grant support from Novartis, Roche, Biogen, Astellas, Aeovian, Bridgebio, Aucta and Quadrant Biosciences. He has served on Scientific Advisory Boards for Roche, Celgene, Regenxbio, Alkermes, and Takeda.