Kishnani, Priya S.
Gibson, James B.
Gambello, Michael J.
Hillman, Richard
Stockton, David W.
Kronn, David
Leslie, Nancy D.
Pena, Loren D.M.
Tanpaiboon, Pranoot
Day, John W.
Wang, Raymond Y.
Goldstein, Jennifer L.
An Haack, Kristina
Sparks, Susan E.
Zhao, Yang
Hahn, Si Houn
Funding for this research was provided by:
Sanofi Genzyme
Article History
Received: 24 January 2019
Accepted: 17 April 2019
First Online: 14 May 2019
Disclosure
: This study was supported by Sanofi Genzyme. Authors employed by Sanofi Genzyme (K.A.H.,S.E.S.; statistician Y.Z. was employed by Sanofi Genzyme when the study was conducted) participated in study design, data collection and interpretation, provided intellectually important revisions to the manuscript, and approved the final version for publication. The authors disclose the following conflicts of interest. From Sanofi Genzyme: S.H.H.: honoraria and travel support (ADVANCE investigators’ meeting); D.K.: research funding; N.D.L.: advisory board honoraria, professional writing support; L.D.M.P.: Pompe and Gaucher advisory boards and Gaucher speakers’ bureau; P.T.: honoraria, travel support as clinical trial principal investigator; J.B.G.: research support, speakers’ bureau; D.W.S.: Pompe Registry Advisory Board, speakers’ bureau, research support, honoraria; R.W.: speaking stipend and travel support, 2014 Pompe Registry meeting; J.L.G., grants during the study; P.S.K.: Pompe and Gaucher Registry advisory boards, research support. From Actus Therapeutics: P.S.K., equity. From Alexion, Amicus, Shire, and Vertex: P.S.K., personal fees. From Baebies, Inc.: P.S.K., advisory board. From Biomarin: P.T., honoraria, travel support as clinical trial principal investigator. From New York Medical College: D.K., research funding. From Roche, Shire, and Valerion: P.S.K., grants. From Seattle Children’s Hospital: S.H.H., personal fees (outside submitted work). The other authors declare no conflicts of interest.