Document is current

Article History

Accepted: 29 October 2024

First Online: 23 November 2024

Declarations

:

: Lauren Schmitt is a consultant for Confluence Pharmaceuticals, Stalicla, and Scioto Biosciences with no direct conflicts to the content presented. Elizabeth Berry-Kravis has received funding from Acadia, Alcobra, AMO, Asuragen, Avexis, Biogen, BioMarin, Cydan, Engrail, Erydel, Fulcrum, GeneTx, GW, Healx, Ionis, Jaguar, Kisbee, Lumos, Marinus, Moment Biosciences, Neuren, Neurogene, Neurotrope, Novartis, Orphazyme/Kempharm/Zevra, Ovid, PTC Therapeutics, Retrophin, Roche, Seaside Therapeutics, Taysha, Tetra, Ultragenyx, Yamo, Zynerba, and Vtesse/Sucampo/Mallinckrodt Pharmaceuticals, to consult on trial design or run clinical or lab validation trials in genetic neurodevelopmental or neurodegenerative disorders, all of which is directed to RUMC in support of rare disease programs; Dr Berry-Kravis receives no personal funds and RUMC has no relevant financial interest in any of the commercial entities listed. David 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. Randall Carpenter is the founder of Seaside Therapeutics, the company that performed the clinical trials. He is also the founder of Allos Pharma, the company that is currently developing arbaclofen for fragile X syndrome. Over the past 24 months, Dejan B. Budimirovic, MD, has received funding from Zynerba Pharmaceuticals (ZYN2-CL-017, ZYN2-CL-033) and Tetra Discovery Partners, Inc. (BPN14770-CNS-204, BPN14770-CNS-301, BPN14770-CNS-302) as an investigator on the listed five clinical trials in individuals with fragile X syndrome (FXS). He has also received funding from CDC (RFA-DD-21–002). All the above funding has been directed to the Kennedy Krieger Institute/the Johns Hopkins Medical Institutions, meaning that Dr. Budimirovic receives no personal funds, and the Institute has no relevant financial interest in any of the commercial entities listed. Paul Wang is an employee of Clinical Research Associates, LLC, a non-profit and formally worked for Seaside Therapeutics. Karen-Walton Bowen has previously worked at Seaside Therapeutics and currently works at Clinical Research Associates, LLC. Craig Erickson is a consultant to Spinogenix, Stalicla, and Scioto Biosciences with no direct conflicts to the content presented.