Funding for this research was provided by:
Received: 6 March 2019
Accepted: 23 May 2019
First Online: 4 June 2019
Compliance with Ethical Standards
: The institution that employs AAP and FP received research grants from Ultragenyx for this and other studies. AAP reports advisory board membership and lectures for Ultragenyx. TOC reports research grants from Ultragenyx and consultancy for Clementia, Inozyme, Pharmacosmos, Kirin, and Lakeside. MLB reports Honoraria from Amgen, Bruno Farmaceutici, and Kyowa Kirin, Grants and/or Speaker Fees from Abiogen, Alexion, Amgen, Bruno Farmaceutici, Eli Lilly, Kyowa Kirin, MSD, NPS, Servier, Shire, and SPA, and consultancy for Alexion, Bruno Farmaceutici, Kyowa Kirin, Servier, and Shire. KB reports Personal Fees from Kyowa Kirin, UCB, Amgen, and Lilly. RC reports Grants and Non-financial Support from Ultragenyx. SJdB reports Research Grants, Advisory Board Membership, and Consultancy for Ultragenyx. RE reports Grants and Personal Fees from Amgen and Alexion, Grants from Department of Health, AstraZeneca, ARUK/MRC Centre for Excellence in Musculoskeletal Ageing Research, National Institute for Health Research, MRC/AZ Mechanisms of Diseases Call, and MRC, Grants, Personal Fees, and Non-financial Support from Immunodiagnostic Systems, Grants and other from National Osteoporosis Society, Grants, Personal Fees, and other from Roche, Personal Fees and other from Eli Lilly, other from European Calcified Tissue Society, IOF CSA, IBMS, and ASBMR, and Personal Fees from D-STAR and GSK Nutrition. YI reports Grants from Kyowa Hakko Kirin Co., Ltd. EAI reports Grants and Advisory Board Membership for Ultragenyx Pharmaceutical, Inc. SI reports Research Grants from Ultragenyx. NI and TK report research Grants from Kyowa Hakko Kirin. MJ reports Research Grants and Advisory Board Membership for Kyowa Kirin. PK reports research grants from Ultragenyx. RK reports Research Grants and Speaking Fees from Kyowa Kirin. FP reports Advisory Board Membership and Lectures for Ultragenyx. PP reports Research Grants from Amgen, Ultragenyx, and Shire, and employment by Ascendis Pharma, Inc. SHR reports support from Ultragenyx, Kyowa Kirin, Lilly, and Amgen/UCB for enrolling patients into clinical trials, and non-financial support from Eli Lilly. YT reports Personal Fees from Kyowa Hakko Kirin Co. Ltd., and Grants and Personal Fees from Chugai Pharmaceutical Co. Ltd. and Teijin Pharma Ltd. TJW reports Research Support, Travel Fees, and Honoraria from Ultragenyx Pharmaceutical, Inc. LZ, CTO, MM, and JSM were employees and stockholders of Ultragenyx Pharmaceutical, Inc. during the study. JSM has a Patent Pending that is jointly owned by Ultragenyx Pharmaceutical, Inc. KI reports Research Grants and Advisory Board Membership for Ultragenyx. HIC, MCS, RL, HT, and HWY have nothing to disclose.
: All procedures performed in human participants were in accordance with the Ethical Standards of the Institutional and/or National Research Committee and with the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.
: Informed consent was obtained from all study participants.