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Authors
Legare, Janet M.
Ingram, David G. https://orcid.org/0000-0002-7285-2857
Pauli, Richard M.
Hecht, Jacqueline T.
Dujmusic, Lorena
Rodriguez-Buritica, David F.
Campbell, Jeffrey W.
Modaff, Peggy
Little, Mary E.
Smid, Cory J.
Serna, Maria E.
Bober, Michael B.
Hoover-Fong, Julie E.
Hashmi, S. Shahrukh
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical (188299)
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Article History
Received: 23 October 2024
Revised: 29 December 2024
Accepted: 15 January 2025
First Online: 23 January 2025
Declarations
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: Institutional Review Board approval for the multi-institutional CLARITY study was obtained from the four participating sites: Johns Hopkins University (JHU) (coordinating site); McGovern Medical School at University of Texas Health (UTH); Nemours Children’s Hospital, Delaware (NCH); and University of Wisconsin (UW). The study was performed in accordance with the ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards.
: Patients signed a locally approved informed consent form if they sought care over the data collection period. Subjects who could not be reached during the data collection period were included in the cohort under a waiver of consent. This process was approved by the local Institutional Review Board at each participating institution (Johns Hopkins University (Baltimore, MD; coordinating site), AI DuPont Hospital for Children (Wilmington, DE), McGovern Medical School at the University of Texas Health (Houston, TX), and University of Wisconsin (Madison, WI).
: This is an investigator‑initiated independent research study supported, in part, by BioMarin, Inc. The data presented in this paper do not pertain to the investigational pharmaceuticals under development by BioMarin, Ascendis, Therachon, or QED. These following competing activities were reviewed and approved by our institutions. J.E.H.‑F. has participated in Advisory Boards sponsored by BioMarin pertaining to achondroplasia. J.E.H.‑F. has been consulted by BioMarin, Alexion, Therachon/Pfizer, QED, Innoskel and NovoNordisk for clinical issues related to achondroplasia and other genetic skeletal conditions and acts as a site principal investigator (PI) for clinical trials for BioMarin, Therachon/Pfizer and QED. M.B.B. is now an employee of Tyra pharmaceuticals. J.T.H. has participated in Advisory Boards pertaining to achondroplasia sponsored by BioMarin. D.R.‑B. has participated in Advisory Boards pertaining to achondroplasia sponsored by BioMarin and has lectured for BioMarin. J.L. is a site PI for Ascendis and QED and is a paid speaker and consultant for BioMarin. The other authors declare no competing interests.
