Beck, Michael
Ramaswami, Uma http://orcid.org/0000-0002-4703-7447
Hernberg-Ståhl, Elizabeth
Hughes, Derralynn A.
Kampmann, Christoph
Mehta, Atul B.
Nicholls, Kathleen
Niu, Dau-Ming
Pintos-Morell, Guillem
Reisin, Ricardo
West, Michael L.
Schenk, Jörn
Anagnostopoulou, Christina
Botha, Jaco
Giugliani, Roberto
Funding for this research was provided by:
Shire, a Takeda company
Takeda Development Center Americas, Inc.
Article History
Received: 15 February 2022
Accepted: 6 June 2022
First Online: 20 June 2022
Declarations
:
: FOS was approved by the ethics institutional review boards of participating centers. Further, this registry was compliant with relevant global and local regulations and best practices: Good Pharmacoepidemiological Practice, Good Research for Comparative Effectiveness principles, and the relevant principles of the International Conference on Harmonisation Good Clinical Practice guidelines were followed as appropriate for an observational registry, consistent with the Declaration of Helsinki.
: Patient consent for publication was not required.
: MB reports honoraria for speaking and/or advisory boards from Sanofi Genzyme and Takeda. He is a member of the FOS Steering Committee. UR reports honoraria for speaking and/or advisory boards from Amicus Therapeutics, Sanofi Genzyme, and Takeda, and research grants from Amicus Therapeutics and Takeda. She is a member of the FOS Steering Committee. EH-S reports no conflict of interest. DAH reports honoraria from Amicus Therapeutics, Freeline Therapeutics, Idorsia, Protalix, Sanofi Genzyme, and Takeda. She is a member of the FOS Steering Committee. CK reports honoraria for speaking and/or advisory boards from Amicus, BioMarin, Gore, and Takeda. He is a member of the FOS Steering Committee. ABM reports honoraria from Amicus Therapeutics, Avrobio, Freeline Therapeutics, Protalix, Sanofi Genzyme, and Takeda. He is a former member of the FOS Steering Committee and past chair of the FOS Steering Committee. KN reports honoraria from Amicus Therapeutics, Sanofi Genzyme, and Takeda. She is a member of the FOS Steering Committee. D-MN reports honoraria and speaker fees from Sanofi Genzyme and Takeda, and research grants from BioMarin, Sanofi Genzyme, and Takeda. He is a member of the FOS Steering Committee. GP-M reports honoraria from Alexion, Amicus, BioMarin, Sanofi Genzyme, and Takeda, and unrestricted grants from Sanofi Genzyme and Takeda to the Vall d’Hebron Research Foundation for funding research on rare diseases. He is a member of the FOS Steering Committee. RR reports honoraria, speaker fees, and consulting fees from Amicus Therapeutics, CSL Behring, Gador, Sanofi Genzyme, Novartis, and Takeda. He is a member of the FOS Steering Committee. MLW reports grants, personal fees, and travel support from Amicus Therapeutics, Idorsia, Protalix, Sanofi Genzyme, and Takeda. He is a member of the FOS Steering Committee. JS is an employee of Takeda Pharmaceuticals International AG. and is a stockholder of Takeda Pharmaceuticals Company Limited. CA is an employee of Takeda Pharmaceuticals International AG and is a stockholder of Takeda Pharmaceuticals Company Limited. JB is an employee of Takeda Pharmaceuticals International AG and is a stockholder of Takeda Pharmaceuticals Company Limited. RG reports honoraria, consulting fees, speaker fees, research funding, and/or travel reimbursement from Abeona, Allievex, Amicus Therapeutics, Avrobio, Azafaros, BioMarin, Chiesi, Denali Therapeutics, Idorsia, Inventiva, Janssen, JCR Pharmaceuticals, Lysogene, Novartis, Paradigm Biopharma, Passage Bio, Protalix, PTC Therapeutics, Regenxbio, Sanofi Genzyme, Sigilon, Sobi, Takeda, and Ultragenyx. He is a member and current chair of the FOS Steering Committee.