Funding Support and Author Disclosures Dr Di Costanzo is a recipient of a research position under cofunding from the European Union REACT-EU, PON Research and Innovation 2014-2020. Dr Pirona is supported by GenomeUp srl through an industrial PhD scholarship. Dr Bini has received personal fees for public speaking from SOBI; has stock options in Eli Lilly, UnitedHealth, Novo Nordisk, Merck, and Thermo Fisher Scientific; and has received grants for meeting participation from Novartis. Dr D’Erasmo has received personal fees for public speaking or consultancy or grant support from Amryt Pharmaceutical, Akcea Therapeutics, SOBI, AuroraBiopharma, Novartis, Amarin, Daiichi-Sankyo, Bayer, Ultragenyx, and Sandoz. Dr Baratta has received personal fees for public speaking from Sandoz. Dr Arca has received research grant support from Amryt Pharmaceutical, Amgen, IONIS, Akcea Therapeutics, Daiichi-Sankyo, Novartis, Pfizer, and Regeneron; has served as a consultant for Amgen, Akcea Therapeutics, Daiichi-Sankyo, and Ultragenyx; and has received fees for lecturing, congress participation, and advisory board participation from Amgen, Amryt Pharmaceutical, Daiichi-Sankyo, Regeneron, Sanofi, Amarin, and Ultragenyx. All other authors have reported that they have no relationships relevant to the contents of this paper to disclose.