About the Authors

Giorgio Tasca

Affiliation Don Carlo Gnocchi Onlus Foundation, Milan, Italy

Mauro Monforte

Affiliation Institute of Neurology, Catholic University School of Medicine, Rome, Italy

Elisabetta Iannaccone

Affiliation Institute of Neurology, Catholic University School of Medicine, Rome, Italy

Francesco Laschena

Affiliation Department of Radiology, Istituto Dermopatico dell'Immacolata, IRCCS, Rome, Italy

Pierfrancesco Ottaviani

Affiliation Department of Radiology, Istituto Dermopatico dell'Immacolata, IRCCS, Rome, Italy

Emanuele Leoncini

Affiliation Institute of Hygiene-Department of Public Health, Catholic University School of Medicine, Rome, Italy

Stefania Boccia

Affiliation Institute of Hygiene-Department of Public Health, Catholic University School of Medicine, Rome, Italy

Giuliana Galluzzi

Affiliation Cellular Biology and Neurobiology Institute, CNR, Rome, Italy

Marco Pelliccioni

Affiliation Unione Italiana Lotta Distrofia Muscolare (UILDM) Rome Section, Rome, Italy

Marcella Masciullo

Affiliation IRCCS Santa Lucia Foundation, Rome, Italy

Roberto Frusciante

Affiliation Neurophysiopathology Unit, Columbus Hospital, Catholic University School of Medicine, Rome, Italy

Eugenio Mercuri

Affiliation Pediatric Neurology Unit, Catholic University School of Medicine, Rome, Italy

Enzo Ricci

ericci@rm.unicatt.it

Affiliation Institute of Neurology, Catholic University School of Medicine, Rome, Italy

Competing Interests

The authors of this manuscript have the following competing interests. EM has served on scientific advisory boards for Acceleron Pharma, Prosensa, and PTC Therapeutics; he receives research support from the European Union, Parent Project NL, SMA Europe, and Italian Telethon; he is receiving funding for trials from GlaxoSmithKline; and he has received funding for trials from Trophos and PTC Therapeutics. The other authors have declared that no competing interests exist. The authors also hereby confirm that what is disclosed in the competing interests section does not alter adherence to PLOS ONE policies on sharing data and materials.

Author Contributions

Conceived and designed the experiments: GT ER. Performed the experiments: GT EI FL PO GG MP M. Masciullo RF ER. Analyzed the data: GT M. Monforte EL SB EM ER. Contributed reagents/materials/analysis tools: GT ER. Contributed to the writing of the manuscript: GT M. Monforte EM ER.