Acknowledgments
We would like Dr. Luigi Francesco Iannone for the creation of the figure.
Declaration of interest
IRCCS ‘G. Gaslini’ is a member of ERN-Epicare. A Riva has received honoraria from Kolfarma, Proveca Pharma, PTC Therapeutics, Jazz Pharmaceuticals, and UCB Pharma; E Russo has received speaker fees or funding from, and has participated in advisory boards for, Angelini Pharma, Eisai, Pfizer, GW Pharmaceuticals, Jazz pharmaceuticals, UCB and Kolfarma; P Striano has received speaking grants from Neuraxpharma, Jazz pharma, BioMarin, Proveca, UCB Pharma. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Reviewer disclosures
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.