Hoover-Fong, Julie E. http://orcid.org/0000-0002-1242-5626
Schulze, Kerry J.
Alade, Adekemi Y.
Bober, Michael B.
Gough, Ethan
Hashmi, S. Shahrukh
Hecht, Jacqueline T.
Legare, Janet M.
Little, Mary Ellen
Modaff, Peggy
Pauli, Richard M.
Rodriguez-Buritica, David F.
Serna, Maria E.
Smid, Cory
Liu, Chengxin
McGready, John
Funding for this research was provided by:
biomarin pharmaceutical
Article History
Received: 22 July 2021
Accepted: 28 November 2021
First Online: 23 December 2021
Declarations
:
: This study was approved by the Institutional Review Boards of Johns Hopkins University, Nemours/AI DuPont Hospital for Children, McGovern Medical School, University of Texas Health, and University of Wisconsin School of Medicine and Public Health. Clinically active patients signed a consent form at their participating site. A waiver of consent was used for data inclusion for subjects lost to followup.
: Not applicable.
: 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, and Therachon/Pfizer for clinical issues related to achondroplasia and other genetic skeletal conditions and acts as a site principal investigator (PI) for clinical trials for BioMarin and Therachon/Pfizer. M.B.B. has participated in Advisory Boards sponsored by BioMarin, consulted with BioMarin, Ascendis, Therachon/Pfizer and QED for clinical issues related achondroplasia as well as acting as a site PI in their clinical trials. He is a member of Alexion’s Speaker’s Bureau (HPP). M.B.B. is a site PI and consultant for MedLife Discoveries (RCDP), and a site PI for Shire (Hunter) and SOBI (Sanfilliopo). 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. J.L. is acting as a site PI for Ascendis and has lectured for BioMarin. The other authors declare no competing interests.