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The authors were not paid for the writing of the manuscript. Medical writing support was provided by Statinfo and was funded by Sanofi. Open access fees for the manuscript were paid by Sanofi. M.Sc., F.D., C.D.V., F.G., A.P., and M.Sp. participated in Advisory Boards and were invited speakers in events sponsored by Sanofi. A.Ba. received honoraria for lectures, advisory boards, meetings, and travel support from Chiesi farmaceutici, Sanofi Genzyme, and Takeda. M.A.D. participated in Advisory Boards and events sponsored by Sanofi and received financial support for newborn screening for lsd in Tuscany. S.F. received an honorarium for scientific consultancy by Sanofi, Orchard, Alexion, Takeda, Immedica Pharma AB. C.G. received honoraria for participation to meetings from Sanofi. F.S. received research grants from Amgen, Biogen Idec, Biomarine, Boehringer-Ingelheim, Celgene, Daiichy Sankyo, Eli Lilly, Genzyme, Janssen Cilag, Gore, Lundbeck, MSD Italia, Nestl\u00e8 Health Science, Novartis, Novo Nordisk, Obi, Pfizer, Roche, Sanofi, Servier, Takeda, Teva; participated in Conferences organized by 3P Solutions, Aristea, Dephaforum, Doctom, Dynamicom, Editree, Emeeting, MA Provider, Medic, OIC, Panacea, RegiaCongressi, Seed, Sics, Sidera, Validinsight and participated in Advisory Boards funded by Accord, Amgen, Allergan, Arvelle Th., Celgene, Eli Lilly, Genzyme, GWpharmaceutical, Galapagos, Janssen Cilag, Novartis, Sanofi, Servier, Takeda. G.T. participated in advisory boards on topics not related to this manuscript in the last two years sponsored by: Eli Lilly; Sanofi; Amgen; Novo Nordisk; Sobi; Gilead; Celgene; Daikii Sankyo. Scientific coordinator of the academic spin-off INSPIRE that carried out observational studies on topics not related to the content of this manuscript by several pharmaceutical companies in the last two years. F.C. participated in advisory boards and meetings sponsored by Sanofi, Chiesi.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"This work is based on a survey of disease experts and no patient was involved. No sensitive data were collected.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Human and animal rights"}},{"value":"All disease experts provided written informed consent by electronic means before participation.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Informed consent"}}]}}