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D.H. received speaker fees from Chiesi. S.B. and F.E. received honorary fees and travel expenses from Recordati and Chiesi. P.G. received speaker and consultancy fees from Horizon, Avrobio, and Eloxx Pharma. H.H. received speaker fees from Chiesi. E.L. received speaker fees and consultancy fees from Chiesi, Recordati, and Novartis. L.P. received consultancy fees from Chiesi. R.T. received consultancy fees from Recordati. D.T. was an advisory board member of Amgen, Novartis and the Cystinosis Research Foundation. F.B. received consultancy fees from Chiesi. G.L. and B.T. were advisory board members of Chiesi. P.G. was an advisory board member of Recordati. The other authors declare no competing interests.","order":1,"name":"Ethics","label":"Competing interests","group":{"name":"EthicsHeading","label":"Ethics"}},{"value":"This content has been made available to all.","name":"free","label":"Free to read"}]}}