Eisengart, Julie B.
Pierpont, Elizabeth I.
Kaizer, Alexander M.
Rudser, Kyle D.
King, Kelly E.
Pasquali, Marzia
Polgreen, Lynda E.
Dickson, Patricia I.
Le, Steven Q.
Miller, Weston P.
Tolar, Jakub
Orchard, Paul J.
Lund, Troy C.
Funding for this research was provided by:
National Institute of Neurological Disorders and Stroke (U54NS065768)
National Center for Advancing Translational Sciences (UL1TR002494)
Sanofi Genzyme (NA)
Children's Cancer Research Fund (CCRF)
Article History
Received: 14 January 2019
Accepted: 15 April 2019
First Online: 25 April 2019
Disclosure
: J.B.E. received honoraria, consulting fees, and/or research support from ArmaGen, Gene Spotlight, Inc., Sangamo, and Sanofi Genzyme, and has done contract work for Shapiro Neuropsychology Consulting LLC. K.E.K. is a consultant for Shire Plc; has received research support from Shire, Sanofi Genzyme, and Alexion Pharmaceuticals, Inc.; and has done previous contract work for Shapiro Neuropsychology Consulting. M.P. is a consultant for Moderna Tx, Inc. and BioMarin Pharmaceuticals, Inc. and has received travel support from BioMarin Pharmaceuticals, Inc. L.E.P. has received honoraria, consulting fees, and/or research support from Sanofi Genzyme, Shire, and BioMarin. P.I.D. receives research support from Biomarin and Genzyme. W.P.M. is an employee of Sangamo Therapeutics, Inc.. P.J.O. has received honoraria, consulting fees, and/or research support from Sanofi Genzyme, Bluebird Bio, and Horizon. T.C.L. has received speaker fees and research support from Sanofi Genzyme. The other authors declare no conflicts of interest.