Document is current

Article History

Accepted: 11 July 2021

First Online: 25 August 2021

Declarations

:

: Ming Liang Chan, Yulan Qi, Kevin Larimore, Anu Cherukuri, Lori Seid, Kala Jayaram, Elena Fisheleva, Jonathan Day, Alice Huntsman Labed and Joshua Henshaw are employees of BioMarin Pharmaceutical Inc. Ravi Savarirayan and Klaus Mohnike have received consulting fees and grants from BioMarin. Melita Irving has received consulting fees from BioMarin. Carlos A. Bacino has received consulting fees, honoraria and grants from BioMarin. Julie Hoover-Fong has received consulting fees from BioMarin, Therachon and Ascendis, and grants from BioMarin. Keiichi Ozono has received consulting fees and honoraria from BioMarin. William R. Wilcox was a consultant to BioMarin and received an honoraria, and was the principal investigator for the clinical trial contract with Emory University. William A. Horton is an inventor on a patent application “Type X collagen assay and methods of use thereof”, submitted by Shriners Hospitals for Children. He has consulted for and/or received speaker honoraria from BioMarin, TherAchon (Pfizer), Ascendis, QED, Relay Therapeutics, Fortress Biotech, OPKO, and Medicell Technologies.

: The studies were conducted in accordance with the provisions of the Declaration of Helsinki. The study protocols were approved by the relevant ethics boards at each site.

: Written informed consent from a parent or legal guardian of each patient was obtained, and assent was obtained from patients, if appropriate, prior to enrolment.

: The de-identified individual participant data that underlie the results reported in this article (including text, tables, figures, and appendices) will be made available together with the research protocol and data dictionaries, for non-commercial, academic purposes. Additional supporting documents may be available on request. Investigators will be able to request access to these data, along with supporting documents, via a website beginning at 6 months and ending 2 years after publication. Data associated with any ongoing development program will be made available within 6 months after approval of the relevant product. Requests must include a research proposal clarifying how the data will be used, including proposed analysis methodology. Research proposals will be evaluated relative to publicly available criteria at the BioMarin website to determine whether access will be given, contingent on execution of a data access agreement with BioMarin Pharmaceutical. For data access requests, see ExternalRef removed.

: Not applicable.

: Not applicable.