脊髓性肌肉萎缩症的流行疾病修饰治疗的时代

从小儿神经科(Giorgia Cora首页tti,核磁共振,交流,M.C.P., M.D.P., E.M.), Università Cattolica del Sacro Cuore, Rome; Centro Clinico Nemo (Giorgia Coratti, M.R., A.C., M.C.P., M.D.P., E.M.), Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome; Department of Neurosciences (A.D.), Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome; The NEMO Center in Milan (V.S.), Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Italy; Center of Translational and Experimental Myology (C.B.), and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, University of Genova, IRCCS Istituto Giannina Gaslini; Department of Clinical and Experimental Medicine (S.M.), University of Messina; AOU Città della Salute e della Scienza di Torino (F.R., T.M.), presidio Molinette e OIRM (SS Malattie Neuromuscolari e SC Neuropsichiatria Infantile), Turin; Department of Neurological Sciences (M.C.), AOU Ospedali Riuniti di Ancona; AOU Pisana (Department of Clinical and Experimental Medicine) (G.S.), Neurology Unit, Pisa; Neurology Unit (E.P.), Azienda Ospedale Padova, Padua; Department of Neurology/Stroke Unit (M.T.), Bolzano Hospital, Trentino-Alto Adige; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMO-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Neurology Unit (Giacomo Comi), Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan; Dino Ferrari Center (Giacomo Comi), Department of Pathophysiology and Transplantation, University of Milan; Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit (R.M.), Milan; Neuroimmunology and Neuromuscular Disorders Unit (L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute for Maternal and Child Health (I.B.), IRCCS, Burlo Garofolo, Trieste; NeuroMuscular Unit (M.G.D.A.), Scientific Institute IRCCS E. Medea, Bosisio Parini, Lecco; Scientific Institute IRCCS “E. Medea” (A.T.), Unit for Severe disabilities in developmental Age and Young Adults (Developmental Neurology and Neurorehabilitation), Brindisi; UOC Clinica Neurologica (V.V.), IRCCS Institute of Neurological Sciences of Bologna, Emilia-Romagna; Metabolic Unit (M.D.), A. Meyer Children's Hospital, Florence; Neurology Unit (I.S.), Azienda Ospedaliero-Universitaria, Policlinico Bari “Amaducci”, Bari; Department of Neurosciences (L.R.), Reproductive Sciences and Odontostomatology, University of Naples Federico II; Department of Neurosciences (A.V.), Pediatric Neurology, Santobono-Pausilipon Children's Hospital, Naples; Neurology Unit (L.V.), Department of Neurosciences, University Hospital Santa Maria della Misericordia, Udine, Friuli-Venezia Giulia; Department of Child Neuropsychiatry (A.B.), Fondazione Istituto Neurologico Nazionale C Mondino Istituto di Ricovero e Cura a Carattere Scientifico, Pavia; Dipartimento di Salute della Donna e del Bambino (C.A.), Università di Padova, Padua; IRCCS Istituto delleScienze Neurologiche di Bologna-UOC Neuropsichiatria Infantile (A.P.); Centro Sclerosi Multipla (M.A.M.), P.O. Binaghi, ASSL Cagliari; Cardiomyology and Medical Genetics Unit (L.P.), Università degli Studi della Campania Luigi Vanvitelli Scuola di Medicina e Chirurgia, Napoli; Section of Neurology (F.B.), Department of Biomedicine, Neuroscience, and Advanced Diagnostics (BiND), University of Palermo; Neurology Department (N.C.), Hospital San Francesco of Nuoro; Department of Neuroscience (M.G.), Mental Health and Sensory Organs (NESMOS), Sapienza University of Rome, Sant'Andrea Hospital; Neuromuscular Omnicentre (NeMO) Trento-Fondazione Serena Onlus (R.Z.), Pergine Valsugana; Pediatric Neurology Unit (D.G.), Pediatric Hospital “Giovanni XXIII”, Bari; Child Neuropsychiatry Unit (S.S.), Paediatric Hospital G Salesi, Ancona; Institute of Experimental Neurology (INSPE), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; National Centre for Rare Diseases (D.T.), Istituto Superiore di Sanità, Rome; and Sezione di Igiene (S.B.), Istituto di Sanità Pubblica, Università Cattolica del Sacro Cuore, Rome, Italy.