Maghnie, Mohamad
Semler, Oliver
Guillen-Navarro, Encarna
Selicorni, Angelo
Heath, Karen E.
Haeusler, Gabriele
Hagenäs, Lars
Merker, Andrea
Leiva-Gea, Antonio
González, Vanesa López
Raimann, Adalbert
Rehberg, Mirko
Santos-Simarro, Fernando
Ertl, Diana-Alexandra
Gregersen, Pernille Axél
Onesimo, Roberta
Landfeldt, Erik
Jarrett, James
Quinn, Jennifer
Rowell, Richard
Pimenta, Jeanne
Cohen, Shelda
Butt, Thomas
Shediac, Renée
Mukherjee, Swati
Mohnike, Klaus http://orcid.org/0000-0001-5113-8657
Funding for this research was provided by:
BioMarin Pharmaceutical
Otto-von-Guericke-Universität Magdeburg
Article History
Received: 24 August 2022
Accepted: 27 February 2023
First Online: 15 March 2023
Declarations
:
: All included patients (or the parents or legally accepted representatives thereof) provided informed written consent and were considered to have the cognitive and linguistic capacities necessary to understand and take part in the study. The study was conducted in compliance with the Declaration of Helsinki and all sites received independent ethics committee approval.
: Not applicable.
: MM: Grant support from Pfizer, Novo Nordisk and Merck Serono and consultancy honoraria and speaker fees from Merck Serono, Novo Nordisk, Pfizer, Sandoz, and BioMarin. OS: Speaker fees from BioMarin. EGN: Has received research support, consultation fees and travel support from BioMarin. AS: Consultancy honoraria from BioMarin. KEH: No conflicts of interest in the context of the current study. GH: No conflicts of interest in the context of the current study. LH: Research grants from Pfizer and BioMarin; consultant and lecture fees from Novo Nordisk and Sanofi. AM: No conflicts of interest in the context of the current study. ALG: ALG and Orthopedic and Trauma Research Group (FA-07), IBIMA Tech Unit, has received fees relationship with clinical trials development by BioMarin and QED Therapeutics. These fees do not pose a conflict of interest in connection with the submitted article. VLG: No conflicts of interest in the context of the current study. AR: No conflicts of interest in the context of the current study. MR: No conflicts of interest in the context of the current study. FSS: Advisory consultation fees from BioMarin. DAE: No conflicts of interest in the context of the current study. PAG: BioMarin honoraria for Steering Committee participation. RO: No conflicts of interest in the context of the current study. EL: No conflicts of interest in the context of the current study. JJ: At the time of the research, JJ was a full time employee of BioMarin and held stocks in the company. JQ: At the time of the research, JJ was a full time employee of BioMarin and held stocks in the company. RR, RS, SC, JMP, TB, SM: Full time employees of BioMarin and hold stocks in the company. KM: Honoraria by BioMarin and QED.