Received: 3 July 2020
Accepted: 26 October 2020
First Online: 6 January 2021
Ethics approval and consent to participate
: The study was approved by the local Institutional Review Board, Partners Human Research Committee (PHRC), prior to patient enrollment and included both a prospective protocol (2017P002437) and a retrospective medical record review protocol (2012P000132). For the retrospective protocol, formal informed consent was waived by the PHRC. For the prospective protocol, return of a completed survey implied consent.
: Not applicable.
: FE is the PI of Bluebird Bio and Minoryx Therapeutics clinical trials. He is a consultant to Ionis, Alnylam, Sanofi Genzyme, Minoryx, and SwanBio Therapeutics. He is the Director of the Third Rock MGH Neuroscience Fellowship. He is the Founder of SwanBio Therapeutics.