Linhart, Aleš http://orcid.org/0000-0002-3372-7850
Dostálová, Gabriela
Nicholls, Kathy
West, Michael L.
Tøndel, Camilla
Jovanovic, Ana
Giraldo, Pilar
Vujkovac, Bojan
Geberhiwot, Tarekegn
Brill-Almon, Einat
Alon, Sari
Chertkoff, Raul
Rocco, Rossana
Hughes, Derralynn
Funding for this research was provided by:
Protalix Biotherapeutics
Article History
Received: 19 January 2023
Accepted: 27 September 2023
First Online: 21 October 2023
Declarations
:
: An Institutional Review Board or Independent Ethics Committee reviewed and approved the study protocol and any amendments prior to their implementation, the informed consent forms, their updates, and any written materials given to patients at each site. All patients provided informed written consent to participate before any study activity was initiated. All procedures were followed in accordance with the ethical principles of the Declaration of Helsinki, in compliance with the approved protocol, Good Clinical Practice guidelines, and applicable regulatory requirements.
: The results presented in this paper have not been published previously in whole or part, except in abstract format.
: Not applicable.
: AL: Received speakers’ fees and consultancy honoraria from Chiesi, Sanofi-Genzyme, Takeda, Avrobio, and Alnylam. GD: Received speakers’ fees and consultancy honoraria from Sanofi, Takeda, Amicus, Chiesi, Greenovation Biotech GmbH. KN: Received grants and personal fees from Sanofi-Genzyme, Takeda; personal fees from Amicus, Idorsia, and Protalix. MW: Received research grants, honoraria, and/or consultant fees from Takeda, Sanofi-Genzyme, Amicus, Protalix, and Idorsia. Dr. West owns IP related to cardiac biomarkers and gene therapy in Fabry disease. CT: Received consultancy honoraria from Sanofi Genzyme, Amicus Therapeutics, Chiesi, and Acelink; participated in studies supported by Sanofi Genzyme, Shire, Protalix, Chiesi, Freeline, and Idorsia. AJ: Received grants from Amicus; honoraria and travel and accommodation from Takeda, Amicus, and Sanofi-Genzyme. PG: The author declares no competing interests. BV: Received speakers’ fees and consultancy honoraria from Sanofi-Genzyme, Takeda, Amicus, Chiesi, and Greenovation Biotech. TG: The author declares no competing interests. EB: Employed by Protalix. SA: Employed by Protalix. RC: Former full-time employee of Protalix Biotherapeutics and current consultant to Protalix Biotherapeutics and Chiesi USA, Inc. RR: Full-time employee of Chiesi Farmaceutici S.p.A. DH: Received honoraria for speaking and advisory boards from Amicus, Takeda, Sanofi-Genzyme, Freeline, and Protalix. Honoraria are administered through UCL consultants and are used in part to support laboratory research.