Funding for this research was provided by:
BioMarin Pharmaceutical (no)
Sanofi Genzyme (no)
National Institutes of Health (NIH R01 DK067859)
Received: 12 November 2019
Accepted: 28 January 2020
First Online: 3 February 2020
Ethics approval and consent to participate
: The study was approved by the ethical committee of National Taiwan University Hospital, Taipei, Taiwan. The clinical information was gathered from the hospital medical records retrospectively.
: Informed consent for publication was obtained from all participants of the study using institutional consent forms.
: YHC and WLH have received consulting and advisor fees from Biomarin and Sanofi, and receive research funding from Biomarin and Sanofi. M. H. Gelb serves as a consultant for PerkinElmer and gets funding from Takeda, Biomarin, Ultrageneyx, The Legacy of Angels Foundation and CureGM1 Foundation. Others report no competing interests.