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Article History

Accepted: 14 March 2024

First Online: 23 April 2024

Declarations

:

: These studies were funded by BioMarin Pharmaceutical Inc. Medical writing assistance was provided by Rebecca Lew, PhD, CMPP, and Celine Vivien, PhD, of ProScribe – Envision Pharma Group, and was funded BioMarin Pharmaceutical Inc. ProScribe’s services complied with international guidelines for Good Publication Practice.

: BioMarin Pharmaceutical Inc. was involved in the study design, data collection, data analysis, and preparation of the manuscript.

: Y.Q., K.L., E.F., A.C., J.D., and J.H.: employees and shareholders of BioMarin Pharmaceutical Inc. M.L.C.: former employee and stockholder of BioMarin Pharmaceutical Inc. D.R.M.: paid consultant for BioMarin Pharmaceutical Inc. R.S. and K.O.: honoraria from BioMarin Pharmaceutical Inc. M.I.: honoraria for consultancy services relating to the subject of this manuscript. C.A.B. and K.M.: no conflicts of interest. J.H.-.F.: institution received funding from BioMarin Pharmaceutical Inc. to execute the study presented in this manuscript. Funds were also received from Pfizer and QED for other achondroplasia-related studies. J.H.-F.: consultant for BioMarin Pharmaceutical Inc., Pfizer, QED, and Sanofi, related to achondroplasia. W.R.W.: local principal investigator for the vosoritide studies at Emory University, funded by a clinical trial contract from BioMarin Pharmaceutical Inc. to Emory University. W.R.W. received honoraria for advisory committee meetings from BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. paid for writing assistance for the manuscript. M.B.B.: employed by the Nemours Foundation, which has received institutional support for research from Ascendis Pharma, BioMarin Pharmaceutical Inc., QED, and Therachon/Pfizer. His institution has received consulting fees from BioMarin Pharmaceutical Inc. and Tyra Biosciences. M.B.B. has received consulting fees from Ascendis Pharma, BioMarin Pharmaceutical Inc., QED, and Therachon/Pfizer. He has received compensation for speaking on behalf of the Alexion speaker’s bureau and from Novo Nordisk and Tyra Biosciences. In the future, he will be employed by Tyra Biosciences.

: The de-identified individual participant data that underlie the results reported in this article (including text, tables, figures, and appendixes) 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 .

: The ethical principles outlined in the Declaration of Helsinki, the US Code of Federal Regulations, and the International Conference on Harmonisation Guideline for Good Clinical Practice E6 were followed during the conduct of the study. The study protocols were approved by relevant ethics review boards and informed consent was obtained from all parents or guardians in writing.

: Informed consent was obtained from all parents or guardians in writing.

: Not applicable.

: Not applicable.

: Y.Q., M.L.C., and J.H. contributed to the conceptualization of the study. Y.Q., E.F., A.C., and J.D. curated the data. Y.Q., D.R.M., and A.C. conducted formal analysis. J.D., R.S., M.I., J.H.-F., K.M., W.R.W., and M.B.B. contributed to the investigation. Y.Q., M.L.C., K.L., C.A.B., and J.H. contributed to the methodology. Y.Q. and M.L.C. took on project administration. Y.Q., M.L.C., K.O., W.R.W., and J.H. provided supervision. K.L. oversaw bioanalytical method development, validation, and sample testing. Y.Q., M.L.C., and D.R.M. were responsible for visualizing the data. All authors provided meaningful and substantial edits and critically reviewed/approved the final version of the manuscript.