Received: 19 July 2019
Accepted: 28 October 2019
First Online: 30 December 2019
Compliance with ethical standards
: Employment: Nina Skuban, Jeffrey P. Castelli, Hjalmar Lagast, and Jay A. Barth (Amicus Therapeutics Inc.); stock ownership or options: Nina Skuban, Jeffrey P. Castelli, Hjalmar Lagast, and Jay A. Barth (Amicus Therapeutics Inc.); research grant: Toyo Ohashi (ARVOBIO Inc., Dainippon Sumitomo Pharma, Sanofi Genzyme, Amicus Therapeutics Inc.) and Noroi Sakai (Sanofi Genzyme, Dainippon Sumitomo Pharma); Honoraria: Toyo Ohashi (Dainippon Sumitomo Pharma) and Norio Sakai (Sanofi Genzyme, Dainippon Sumitomo Pharma). All other authors have declared that no conflict of interest exists.
: All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee (IRB approval number for AT1001-012 and AT1001-042: The Jikei University School of Medicine: 23-25(2845) and 26-07(2935); Osaka City University Graduate School of Medicine: 1688 and 1827; Osaka University Graduate School of Medicine: 124002 and 146062; Niigata University Graduate School of Medical and Dental Sciences: CH23-017 and CH26-013) at which the studies were conducted according to International Conference on Harmonisation/Good clinical Practice (ICH/GCP) and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
: Informed consent was obtained from all participants included in the present study.