Quijada-Fraile, Pilar http://orcid.org/0000-0003-1026-4854
Arranz Canales, Elena
Martín-Hernández, Elena
Ballesta-Martínez, María Juliana
Guillén-Navarro, Encarna
Pintos-Morell, Guillem
Moltó-Abad, Marc
Moreno-Martínez, David
García Morillo, Salvador
Blasco-Alonso, Javier
Couce, María Luz
Gil Sánchez, Ricardo
Cortès-Saladelafont, Elisenda
López Rodríguez, Mónica A.
García-Silva, María Teresa
Morales Conejo, Montserrat
Funding for this research was provided by:
BioMarin Pharmaceuticals España SL
Article History
Received: 19 February 2021
Accepted: 10 October 2021
First Online: 3 November 2021
Declarations
:
: The study was approved by the Ethics Committee of the University Hospital12 de Octubre, Madrid. In addition, the study was conducted in accordance with the Declaration of Helsinki [CitationRef removed], the International Conference on Harmonisation Guideline for Good Clinical Practice [CitationRef removed], and the Spanish regulations. All patients were given information about the study both verbally and in writing, and all gave informed consent before entering the study.
: Not applicable.
: PQF, EMH, EAC, SGM, JBA, RGS, ECS, MTGS and MALR have no competing interests. MMC received honoraria for lectures from the Takeda Pharmaceutical Company (Takeda), Sanofi Genzyme, Vitaflo International (Vitaflo), and BioMarin Pharmaceutical Inc. (BioMarin). GPM received honoraria for lectures and/or consulting and travel support from Takeda, Sanofi, BioMarin, Amicus Therapeutics (Amicus), Kyowa Kirin International, Alexion Pharmaceuticals (Alexion) and Vitaflo. MMA received research support from Takeda, Sanofi Genzyme, Amicus, Kaleido, and Vitaflo, and fees from Sanofi Genzyme, Takeda, Alexion, and Amicus for participation in their respective registries. EGN received research and meeting support from BioMarin, Sanofi Genzyme, Takeda, and Alnylam Pharmaceuticals. DMM received travel grants from Amicus, Sanofi and Takeda.