Competing interests
P.N.: Medical writing support for the manuscript as declared in the Acknowledgments; consulting fees from Amicus, Chiesi, Greenovation, Idorsia, Sanofi-Genzyme, Takeda; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from Amicus, Chiesi, Greenovation, Idorsia, Sanofi-Genzyme, Takeda; participation on a data safety monitoring board or advisory board for Amicus, Chiesi, Greenovation, Idorsia, Sanofi-Genzyme, Takeda. O.G.-A.: Medical writing support for the manuscript as declared in the Acknowledgments; grant support from Idorsia, Sanofi, Protalix, Chiesi, Sangamo, 4DMT for clinical research trials; grant support from Sanofi and Takeda for other investigator-initiated studies; has/will receive(d) consulting fees from Sanofi, Chiesi, Takeda, Uniqure; payment or honoraria for speaker bureaus for Sanofi and Takeda; participation on an advisory board on Fabry disease for Chiesi and Sanofi. J.B.: Medical writing support for the manuscript as declared in the Acknowledgments; research support for clinical trial from Idorsia Pharmaceuticals; research support from AVROBIO, BioMarin Pharmaceutical, Chiesi Farmaceutici, Pfizer, Protalix BioTherapeutics, Sangamo Therapeutics, Sanofi, Takeda, Travere Therapeutics; consulting fees from Chiesi USA and Takeda for advisory boards; speaker honorarium from Fabry Support and Information Group. D.P.G.: Medical writing support for the manuscript as declared in the Acknowledgments; consulting fees from Chiesi, Idorsia, Sanofi, and Takeda; support for attending meetings and/or travel from Chiesi, Sanofi, and Takeda. P.G.: Medical writing support for the manuscript as declared in the Acknowledgments. A.J.: Medical writing support for the manuscript as declared in the Acknowledgments; research grants from Takeda and Amicus; payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from Amicus, Chiesi, Takeda, and Sanofi; support for attending meetings and/or travel from Amicus and Chiesi. V.K.: Medical writing support for the manuscript as declared in the Acknowledgments; payment for expert testimony from Sanofi; participation on a Pompe disease advisory board for Sanofi; PI of trials for Protalix and Idorsia. K.N.: Medical writing support for the manuscript as declared in the Acknowledgments; research/clinical trial funding to institution from Sanofi, Takeda, Amicus and Idorsia; payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing or educational events from Takeda, Sanofi and Amicus; participation on a data safety monitoring board or advisory board for Takeda, Sanofi and Amicus. C.R.G.: Medical writing support for the manuscript as declared in the Acknowledgments. R.S.: Medical writing support for the manuscript as declared in the Acknowledgments. M.T.: Medical writing support for the manuscript as declared in the Acknowledgments. A.T.-S.: Medical writing support for the manuscript as declared in the Acknowledgments. E.W.: Medical writing support for the manuscript as declared in the Acknowledgments; grants or contracts from Sanofi, Idorsia, Chiesi, Protalix, Walking Fish Therapeutics, Spark Therapeutics; consulting fees from Sanofi, Amicus, Chiesi, Protalix, Walking Fish Therapeutics, Spark Therapeutics; payment or honoraria for lectures, presentations, speaker bureaus, manuscript writing, or educational events from Sanofi, Chiesi, and Natera; support for attending meetings and/or travel from Sanofi, Chiesi, and Protalix. R.W.: Medical writing support for the manuscript as declared in the Acknowledgments; employed by Idorsia (the study funder) during the planning and execution of the study; owns stock in Idorsia. M.W.: Medical writing support for the manuscript as declared in the Acknowledgments; contract for research from Chiesi; grants from Takeda, Sanofi and Amicus; IP for Fabry gene therapy and Fabry cardiac biomarkers; consulting fees from Takeda; honoraria for CME presentations from Takeda, Sanofi and Sumitomo; payment for expert testimony from Takeda and Amicus; support for travel expenses from Amicus; participation on advisory boards for Sanofi and Amicus; Chair of the Scientific Committee for the CFDI Registry; Member of the Fabry Outcome Survey Steering Committee for Takeda; Member of the Scientific Committee for the Canadian Symposium on Lysosomal Diseases; Member of the North American Advisory Board for Sanofi; Member of the Scientific Committee for the Fabry Update Meeting. M.C.: Medical writing support for the manuscript as declared in the Acknowledgments; employee of Idorsia Pharmaceuticals Ltd; shareholder of Idorsia Pharmaceuticals Ltd. A.F.: Medical writing support for the manuscript as declared in the Acknowledgments; employee of Idorsia Pharmaceuticals Ltd; shareholder of Idorsia Pharmaceuticals Ltd. L.T.: Medical writing support for the manuscript as declared in the Acknowledgments; employee of Idorsia Pharmaceuticals (Sponsor) at the time of data generation, statistical evaluation, and data interpretation; shareholder of Idorsia Pharmaceuticals Ltd. M.M.: Medical writing support for the manuscript as declared in the Acknowledgments; employee of Idorsia Pharmaceuticals Ltd; owns stock. M.V.: Medical writing support for the manuscript as declared in the Acknowledgments; employee of Idorsia Pharmaceuticals Ltd; shareholder of Idorsia Pharmaceuticals Ltd. C.W.: Medical writing support for the manuscript as declared in the Acknowledgments; grant(s) from Boehringer Ingelheim and Sanofi; consultancy fees from Amgen, Amicus, Astellas, AstraZeneca, Bayer, Boehringer Ingelheim, Vifor Pharma, Chiesi, Chugai, Fresenius Medical Care, GSK, Idorsia, Eli Lilly, MSD, Novartis, Novo Nordisk, Sanofi. D.H.: Medical writing support for the manuscript as declared in the Acknowledgments; consultancy fees for advisory boards from Idorsia, Amicus, Sanofi, Takeda, Chiesi, Freeline, Sangamo; honoraria for speaking from Idorsia, Amicus, Sanofi, Takeda, Chiesi, Freeline, Sangamo; support for attending meetings and/or travel from Amicus, Sanofi, Freeline, Chiesi.