Funding for this research was provided by:
Fondazione Telethon (GSP 13002)
Received: 30 December 2019
Accepted: 17 March 2020
First Online: 5 April 2020
Ethics approval and consent to participate
: Approval to conduct this study was received by the ethical committees of the participating centres.
: Not applicable.
: Eugenio Mercuri has received funding as a member of advisory boards for SMA studies for AveXis, Biogen, Ionis Pharmaceuticals, Inc., Novartis, Scholar Rock and Roche; principal investigator for ongoing Ionis Pharmaceuticals, Inc./Biogen, Avexis and Roche clinical trials; funding from Famiglie SMA Italy, Italian Telethon, Biogen and SMA Europe.Dr. Giorgia Coratti received funding from Biogen, Roche, Avexis and Genesis Pharma as speaker in sponsored symposia.Dr. Roberto de Sanctis received funding from Biogen and Avexis as speaker in sponsored symposia.Dr. Maria Carmela Pera received funding from Roche as speaker in sponsored symposia.Dr. Marika Pane received funding as a member of advisory boards from Biogen and Avexis and as speaker in sponsored symposia.Dr. Jacqueline Montes receives support from the Eunice Kennedy Shriver National Institute for Child Health and Human Development (NICHD) 1K01HD084690-01A1 and reports serving as a consultant for Ionis pharmaceuticals and Biogen, and serves on advisory boards for Roche Pharmaceuticals and Biogen.Dr. Darryl C. de Vivo has received personal compensation for activities with AveXis, Biogen, Roche, IONIS, Sarepta, Cytokinetics Pharmaceuticals, and the SMA Foundation and has received research support from NIH, DOD, SMA Foundation, and Hope for Children Research Foundation.Dr. Basil T. Darras has been a scientific advisory board consultant for AveXis, Biogen, Cytokinetics, Marathon, PTC, Roche, and Sarepta; advisor for Ionis Pharmaceuticals, Inc.; research support from the National Institutes of Health/National Institute of Neurological Disorders and Stroke, the Slaney Family Fund for SMA, and the SMA Foundation; grants from Ionis Pharmaceuticals, Inc., Biogen, Cytokinetics, Fibrogen, PTC, Sarepta, and Summit.Dr. Stephen J. Kolb has been a scientific advisory board consultant for AveXis, Genentech and Biogen.Dr. Richard S. Finkel Richard Finkel has received personal compensation for activities with Ionis Pharmaceuticals, Biogen, AveXis, Capricor, Catabasis, Lilly, Roche, Novartis; and the SMA Foundation, SMA Europe and Cure SMA as a consultant or advisor. Dr. Finkel has received research support from Ionis Pharmaceuticals, Biogen, Lilly, Cytokinetics Sarepta, NIH, MDA, and Summit.Dr. Simona Lucibello, Dr. Marco Perulli have nothing to disclose.