About the Authors
- Marika Pane
-
Contributed equally to this work with: Marika Pane, Elena Stacy Mazzone, Serena Sivo
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Elena Stacy Mazzone
-
Contributed equally to this work with: Marika Pane, Elena Stacy Mazzone, Serena Sivo
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Serena Sivo
-
Contributed equally to this work with: Marika Pane, Elena Stacy Mazzone, Serena Sivo
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Maria Pia Sormani
-
Affiliation Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy
- Sonia Messina
-
Affiliation Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy
- Adele D′Amico
-
Affiliation Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy
- Adelina Carlesi
-
Affiliation Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy
- Gianluca Vita
-
Affiliation Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy
- Lavinia Fanelli
-
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Angela Berardinelli
-
Affiliation Child Neurology and Psychiatry Unit, “Casimiro Mondino” Foundation, Pavia, Italy
- Yvan Torrente
-
Affiliation Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Valentina Lanzillotta
-
Affiliation Neuromuscular Disease Unit, Giannina Gaslini Institute, Genoa, Italy
- Emanuela Viggiano
-
Affiliation Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy
- Paola D′Ambrosio
-
Affiliation Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy
- Filippo Cavallaro
-
Affiliation Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Messina, Italy
- Silvia Frosini
-
Affiliation Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy
- Andrea Barp
-
Affiliation Department of Neurosciences, University of Padua, Padua, Italy
- Serena Bonfiglio
-
Affiliation Child Neurology and Psychiatry Unit, Maggiore Hospital, Bologna, Italy
- Roberta Scalise
-
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Roberto De Sanctis
-
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Enrica Rolle
-
Affiliation Neuromuscular Center, San Giovanni Battista Hospital, University of Turin, Turin, Italy
- Alessandra Graziano
-
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Francesca Magri
-
Affiliation Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Concetta Palermo
-
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
- Francesca Rossi
-
Affiliation Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy
- Maria Alice Donati
-
Affiliation Metabolic and Neuromuscular Unit, Meyer Hospital, Florence, Italy
- Michele Sacchini
-
Affiliation Metabolic and Neuromuscular Unit, Meyer Hospital, Florence, Italy
- Maria Teresa Arnoldi
-
Affiliation Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy
- Giovanni Baranello
-
Affiliation Developmental Neurology Unit, Neurological Institute Carlo Besta, Milan, Italy
- Tiziana Mongini
-
Affiliation Neuromuscular Center, San Giovanni Battista Hospital, University of Turin, Turin, Italy
- Antonella Pini
-
Affiliation Child Neurology and Psychiatry Unit, Maggiore Hospital, Bologna, Italy
- Roberta Battini
-
Affiliation Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy
- Elena Pegoraro
-
Affiliation Department of Neurosciences, University of Padua, Padua, Italy
- Stefano Previtali
-
Affiliation Department of Neurology, San Raffaele Scientific Institute, Milan, Italy
- Claudio Bruno
-
Affiliation Neuromuscular Disease Unit, Giannina Gaslini Institute, Genoa, Italy
- Luisa Politano
-
Affiliation Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy
- Giacomo P. Comi
-
Affiliation Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
- Enrico Bertini
-
Affiliation Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy
- Eugenio Mercuri
-
* E-mail: mercuri@rm.unicatt.it
Affiliation Department of Paediatric Neurology, Catholic University, Rome, Italy
Competing Interests
Dr. Pane, Dr. Sivo and E. Mazzone report no disclosures. Dr. Sormani: serves on a scientific advisory board for Biogen Idec.; has received funding for travel or speaker honoraria from Merck Serono; serves as consultant for Merck Serono, Actelion Pharmaceuticals Lts. Biogen Idec, and Synthon; and serves on the speakers' bureaus of Teva Pharmaceutical Industries Ltd., Merck Serono, and Biogen Idec. Dr. D'Amico and Dr. Messina report no disclosures. Dr. Vita serves as an Associate Editor for Neurological Sciences. L. Fanelli, Dr. Berardinelli, Dr. Torrente, V. Lanzillotta, Dr. Viggiano, Dr. D'Ambrosio, F. Cavallaro, S. Frosini, Dr. Barp, Dr. Bonfiglio, Dr. Scalise, R. De Sanctis, E. Rolle, Dr. Graziano, Dr. Magri, Dr. Palermo, F. Rossi, Dr. Donati, Dr. Sacchini, Dr. Arnoldi, Dr. Baranello report no disclosures. Dr. Mongini has served on a scientific advisory board for Telethon Italy; has received funding for travel from Genzyme Corporation; and has received research support from AIFA (Italian Government Drug Agency) and Telethon Italy. Dr. Pini, Dr. Battini report no disclosures. Dr. Pegoraro has served on a scientific advisory board for BioMarin Pharmaceutical Inc.; has received funding for travel from Genzyme Corporation; and has received speaker honoraria from MedaPharma; and receives research support from Wellstone and Telethon Italy. Dr. Previtali, Dr. Bruno, Dr. Politano report no disclosures. Dr. Comi: is site PI for the PTC extension study of Ataluren in DMD, for the GSK study on exon skipping; receives research support from Telethon Italy and SMA Europe. Dr. Bertini is site PI for the PTC extension study of Ataluren in DMD, for the GSK study on exon skipping. He also receives funds from the Italian Telethon, the Italian Ministry of Health and SMA Europe for observational studies on outcome measures. Dr. Mercuri is site PI for the PTC extension study of Ataluren in DMD, for the GSK Prosensa and Sarepta studies on exon skipping. He also receives funds from the Italian Telethon and SMA Europe. He has acted as advisory board for Acceleron Pharma, Shire and PTC Therapeutics, Inc, Prosensa. The disclosures shown in the paper do not alter the authors' adherence to PLOS ONE policies on sharing data and materials.
Author Contributions
Conceived and designed the experiments: MP ESM SS LF RDS EM. Performed the experiments: SM LF RDS VL FC ER SF MTA FR. Analyzed the data: MP ESM SS MPS SM AD AC GV AB YT VL EV PD FC SF AB SB CP RS RDS ER AG FM MAD MS MTA FR GB TM AP RB EP SP CB LP GPC EB EM. Wrote the paper: MP ESM SS MPS SM AD AC GV LF AB YT VL EV PD FC SF AB SB CP RS RDS ER AG FM MAD MS MTA FR GB TM AP RB EP SP CB LP GPC EB EM.