Funding for this research was provided by:
Foundation for Angelman Syndrome Therapeutics (FAST Trac 2018-001)
Received: 11 August 2019
Accepted: 13 December 2019
First Online: 9 January 2020
Ethics approval and consent to participate
: This clinical trial protocol was approved by the IRB at Vanderbilt University in November 2018 (protocol number DUISJ10212017084407). Potential parents/guardians of participants will be contacted initially by a member of the Angelman syndrome clinic at Vanderbilt University. Parents will be provided with the IRB approved version of the informed consent document (Additional fileInternalRef removed). Participants are children between the ages of 4 to 11 years and, therefore, consent obtained from the person with parental rights after they have read the informed consent and had their questions answered.
: Consent is not applicable; the publication plan is in Additional fileInternalRef removed.
: DLH is employed by Disruptive Enterprises, LLC which has exclusively licensed the patents to the formula used in this study. EJW is an inventor on one of the patents covering the study formulation, has received research support from the Foundation for Angelman Syndrome Therapeutics, and is currently employed by PTC Therapeutics which engages in the development and commercialization of gene therapies for Angelman syndrome. DPD is an inventor on one of the patents covering the study formulation, and has received funding from Disruptive Enterprises, LLC and the Office of Naval Research. JD has received funding from the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation.