Semler, Oliver http://orcid.org/0000-0003-0029-7556
Cormier-Daire, Valérie
Lausch, Ekkehart
Bober, Michael B. http://orcid.org/0000-0002-6178-1264
Carroll, Ricki http://orcid.org/0000-0002-9221-4879
Sousa, Sérgio B. http://orcid.org/0000-0003-3921-1334
Deyle, David
Faden, Maha
Hartmann, Gabriele
Huser, Aaron J.
Legare, Janet M. http://orcid.org/0000-0001-6684-4909
Mohnike, Klaus
Rohrer, Tilman R. http://orcid.org/0000-0001-9516-7863
Rutsch, Frank
Smith, Pamela http://orcid.org/0000-0002-2588-9657
Travessa, Andre M. http://orcid.org/0000-0002-4147-2800
Verardo, Angela
White, Klane K.
Wilcox, William R.
Hoover-Fong, Julie http://orcid.org/0000-0002-1242-5626
Funding for this research was provided by:
BioMarin Pharmaceutical
Universitätsklinikum Köln
Article History
Received: 1 June 2023
Accepted: 3 October 2023
First Online: 26 October 2023
Declarations
:
: In addition to the individual conflict of interest outlined below, all authors participated in the expert group meetings, sponsored and organized by BioMarin Pharmaceutical Inc, on which this manuscript is based. Oliver Semler: has received grants or contracts from Kyowa Kirin, payment or honoraria for lectures/presentations from BioMarin and Kyowa Kirin, and support for attending meetings and/or travel from Pfizer. Valérie Cormier-Daire: has received payment or honoraria for lectures/presentations from BioMarin and Ipsen, and participated in Advisory Board meetings for BioMarin, QED-Propel and Mereo. Ekkehart Lausch: has received grants or contracts from DFG (SFB1453), H2020, consulting fees, support for attending meetings and/or travel, and has participated in Advisory Board meetings for BioMarin. Michael B. Bober: has received grants or contracts from BioMarin, Ascendis, QED and Pfizer (all Institutional), consulting fees from BioMarin and Tyra (Institutional), Ascendis and Bridge Bio, payment or honoraria for lectures/presentations from NovoNordisk and Tyra, has participated in Advisory Board meetings for BioMarin, and has a leadership role for the Skeletal Dysplasia Management Consortium. Ricki Carroll: has received grants or contracts from BioMarin, QED, Ascendis and Pfizer (all Institutional), and consulting fees from Guidepoint. Sérgio B. Sousa: has received payment or honoraria for lectures/presentations, support for attending meetings and/or travel, and has participated in Advisory Board meetings for BioMarin, and has a leadership role for the European Achondroplasia Forum and ERN-BOND. David Deyle: has received grants or contracts from Castle Creek. Maha Faden: has received support for attending meetings and/or travel, and has participated in Advisory Board meetings for BioMarin. Gabriele Hartmann: has received consulting fees, support for attending meetings and/or travel, and has participated in Advisory Board meetings for BioMarin. Aaron J. Huser: has received payment or honoraria for speaker bureau, has participated in Advisory Board meetings for BioMarin, and holds a leadership role for the MHE Research Foundation. Janet M. Legare: has received grants or contracts for clinical trials from Ascendis Pharma and Bridge Bio, and for a natural history study from BioMarin, and payment or honoraria for lectures/presentations from BioMarin. Klaus Mohnike: has received grants or contracts for clinical trials, payment or honoraria for lectures/presentations, support for attending meetings and/or travel and has participated in Advisory Board meetings for BioMarin. Tilman R. Rohrer: has received grants or contracts, consulting fees, payment or honoraria for lectures/presentations and support for attending meetings and/or travel from BioMarin and NovoNordisk. Frank Rutsch: has received grants or contracts (Institutional), consulting fees, payment or honoraria for lectures/presentations, support for attending meetings and/or travel, and has participated in Advisory Board meetings for BioMarin. Pamela Smith: has received payment or honoraria for lectures/presentations, and support for attending meetings and/or travel from BioMarin. Andre M. Travessa: has received consulting fees, support for attending meetings and/or travel, and participated in Advisory Board meetings. Angela Verardo: Has received payment or honoraria for speaker bureau from BioMarin. Klane K. White: Has received grants or contracts from BioMarin, Ascendis, Ultragenyx and Pfizer, royalties or licenses for UptoDate.com, payment or honoraria for lectures/presentations from BioMarin and has a leadership role for Little People of America, National MPS Society, Pediatric Orthopedic Society of North America, Scoliosis Research Society, Camp Korey, and the Skeletal Dysplasia Management Consortium. William R. Wilcox: has received grants or contracts (Institutional), and has participated in Advisory Board meetings for BioMarin. Julie Hoover-Fong: has received grants or contracts for clinical trials from BioMarin, QED and Pfizer, consulting fees from BioMarin, QED, NovoNordisk and Innoskel, payment or honoraria for lectures/presentations from Medscape and BioMarin, has participated in Advisory Board meetings for MCDS—Therapy Clinical Trial (unpaid), BioMarin and QED, and is an unpaid member of the Skeletal Dysplasia Management Consortium and of the Medical Advisory Board of the Little People of America.
: This article is based on previously conducted studies and does not contain any new studies with human participants or animals performed by any of the authors.