Kolevzon, Alexander http://orcid.org/0000-0001-8129-2671
Delaby, Elsa
Berry-Kravis, Elizabeth http://orcid.org/0000-0001-7099-1522
Buxbaum, Joseph D. http://orcid.org/0000-0001-8898-8313
Betancur, Catalina http://orcid.org/0000-0002-3327-4804
Funding for this research was provided by:
National Institute of Neurological Disorders and Stroke (R01NS105845-01)
New York Community Trust Jules and Ethel Klein Fund ((none))
Beatrice and Samuel A. Seaver Foundation ((none))
NIH Rare Disease Clinical Research Network (1 U54 NS092090-01)
Article History
Received: 2 September 2019
Accepted: 9 October 2019
First Online: 24 December 2019
Ethics approval and consent to participate
: Not applicable
: Not applicable
: AK receives research support from AMO Pharma and consults to Ovid Therapeutics, Coronis Neurosciences, LabCorp, sema4, and Takeda. EBK has received funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren, Cydan, Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Ovid, Yamo, Acadia, Ionis, Ultragenyx, and Lumos Pharmaceuticals to consult on trial design or development strategies and/or conduct clinical studies in fragile X syndrome or other neurologic, neurodevelopmental, or neurodegenerative disorders; from Vtesse/Sucampo/Mallinckrodt Pharmaceuticals to conduct clinical trials in Niemann-Pick disease type C; and from Asuragen Inc. to develop testing standards for <i>FMR1</i> testing. All funding to EBK is directed to Rush University Medical Center in support of rare disease programs. JDB and Mount Sinai Hospital hold a shared patent for the use of insulin-like growth factor-1 in Phelan-McDermid syndrome; JDB consults with Coronis Neurosciences and sema4. AK, EBK, and CB are on the advisory board of the Phelan-McDermid Syndrome Foundation. ED declares that she has no competing interests.