부산대학교 도서관

Gervasoni, J; Primiano, A; Marini, F; Sabino, A; Biancolillo, A; Calvani, R; Picca, A; Marzetti, E; Persichilli, S; Urbani, A; Servidei, S; Primiano, G

Genes. 11(12)

Montano, V.; Lopriore, P.; Gruosso, F.; Carelli, V.; Comi, G. P.; Filosto, M.; Lamperti, C.; Mongini, T.; Musumeci, O.; Servidei, S.; Tonin, P.; Toscano, A.; Primiano, G.; Valentino, M. L.; Bortolani, S.; Marchet, S.; Ricci, G.; Modenese, A.; Cotti Piccinelli, S.; Risi, B.; Meneri, M.; Arena, I. G.; Siciliano, G.; Mancuso, Michelangelo

Journal of Neurology. 269(12):6555-6565

Nneka Southwell; Guido Primiano; Viraj Nadkarni; Nabeel Attarwala; Emelie Beattie; Dawson Miller; Sumaitaah Alam; Irene Liparulo; Yevgeniya I Shurubor; Maria Lucia Valentino; Valerio Carelli; Serenella Servidei; Steven S Gross; Giovanni Manfredi; Qiuying Chen; Marilena D'Aurelio

EMBO Molecular Medicine, Vol 15, Iss 7, Pp n/a-n/a (2023)

Montano, V.; Orsucci, D.; Carelli, V.; La Morgia, C.; Valentino, M. L.; Lamperti, C.; Marchet, S.; Musumeci, O.; Toscano, A.; Primiano, G.; Santorelli, F. M.; Ticci, C.; Filosto, M.; Rubegni, A.; Mongini, T.; Tonin, P.; Servidei, S.; Ceravolo, R.; Siciliano, G.; Mancuso, Michelangelo

Journal of Neurology. 269(3):1413-1421

Hathazi, D; Griffin, H; Jennings, MJ; Giunta, M; Powell, C; Pearce, SF; Munro, B; Wei, W; Boczonadi, V; Poulton, J; Pyle, A; Calabrese, C; Gomez-Duran, A; Schara, U; Pitceathly, RD; Hanna, MG; Joost, K; Cotta, A; Paim, JF; Navarro, MM; Duff, J; Mattmann, A; Chapman, K; Servidei, S; Della Marina, A; Uusimaa, J; Roos, A; Mootha, V; Hirano, M; Tulinius, M; Giri, M; Hoffmann, EP; Lochmuller, H; DiMauro, S; Minczuk, M; Chinnery, PF; Muller, JS; Horvath, R

The EMBO journal. 39(23):e105364

Bortolani S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Savarese M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Vattemi G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Bonanno S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Falzone YM; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Pugliese A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Primiano G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Sancricca C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Lopergolo D; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Greco G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Gemelli C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ravaglia S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Bencivenga RP; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Velardo D; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Magri F; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Valentino ML; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Cheli M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Torchia E; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Lucchini M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Petrucci A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ricci G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Garibaldi M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Astrea G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Rubegni A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Angelini CI; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ariatti A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Santorelli FM; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ruggieri A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Antonini G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Siciliano G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Filosto M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Mirabella M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Liguori R; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Comi GP; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ruggiero L; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Grandis M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Massa R; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Malandrini A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Servidei S; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Mongini TE; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Rodolico C; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Toscano A; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Previtali SC; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Tonin P; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Diaz-Manera J; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Monforte M; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Ricci E; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Maggi L; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.; Tasca G; From the UOC di Neurologia (S. Bortolani, G.P., C.S., M.L., M. Mirabella, S.S., M. Monforte, E.R., G.T.), Fondazione Policlinico Universitario 'A. Gemelli' IRCCS, Rome; Department of Neurosciences Rita Levi Montalcini (S. Bortolani, T.E.M.), University of Torino, Italy; Folkhälsan Research Center (M.S.), Helsinki, Finland; Department of Neurosciences, Biomedicine and Movement Sciences (G.V., P.T.), University of Verona; Neuroimmunology and Neuromuscular Disorders Unit (S. Bonanno, M.C., A. Ruggieri, L.M.), Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan; Institute of Experimental Neurology (INSPE) (Y.M.F., S.C.P.), Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan; ERN-NMD Center for Neuromuscular Disorders of Messina (A. Pugliese, C.R., A.T.), Department of Clinical and Experimental Medicine, University of Messina; Fondazione UILDM Lazio Onlus (G.P., C.S.), Rome; Department of Medicine, Surgery and Neurosciences (D.L., A.M.), University of Siena; Neuromuscular Diseases Unit (G.G., R.M.), Department of Systems Medicine, University of Rome Tor Vergata, Rome; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal Infantile Sciences (C.G., M. Grandis), University of Genoa; IRCCS Ospedale Policlinico San Martino (C.G., M. Grandis), Genova; IRCCS Mondino Foundation (S.R.), Pavia; Dipartimento di Neuroscienze e Scienze Riproduttive ed Odontostomatologiche (R.P.B., L.R.), Università degli Studi di Napoli 'Federico II,' Naples; Neuromuscular and Rare Diseases Unit (D.V.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico; Neurology Unit (F.M., G.P.C.), Department of Neuroscience and Mental Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; IRCCS Institute of Neurological Sciences of Bologna (M.L.V., R.L.), UOC Clinica Neurologica; Department of Biomedical and NeuroMotor Sciences (M.L.V., R.L.), University of Bologna; Università Cattolica del Sacro Cuore (E.T., M. Mirabella, S.S., E.R.); Center for Neuromuscular and Neurological Rare Diseases S. Camillo Forlanini Hospital (A. Petrucci), Rome; Department of Clinical and Experimental Medicine (G.R., G.S.), Neurological Clinic, University of Pisa; Neuromuscular and Rare Disease Center (M. Garibaldi, G. Antonini), Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), SAPIENZA University, Sant'Andrea Hospital, Rome; Molecular Medicine for Neurodegenerative and Neuromuscular Diseases Unit (G. Astrea, A. Rubegni, F.M.S.), IRCCS Stella Maris Foundation, Pisa; Department of Neurosciences (C.I.A.), University of Padova; Department of Neurosciences (A.A.), University Hospitals of Modena; Department of Clinical and Experimental Sciences (M.F.), University of Brescia; NeMo-Brescia Clinical Center for Neuromuscular Diseases (M.F.), Brescia; Department of Pathophysiology and Transplantation (G.P.C.), Dino Ferrari Center, University of Milan, Italy; and John Walton Muscular Dystrophy Research Centre (J.D.-M., G.T.), Newcastle University and Newcastle Hospitals NHS Foundation Trusts, Newcastle Upon Tyne, United Kingdom.

Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE

Lillo R; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy; Department of Cardiovascular and Pulmonary Sciences, Università Cattolica del Sacro Cuore, Rome, Italy.; Meucci MC; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Malara S; Department of Cardiovascular and Pulmonary Sciences, Università Cattolica del Sacro Cuore, Rome, Italy.; Primiano G; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Servidei S; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Lombardo A; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy; Department of Cardiovascular and Pulmonary Sciences, Università Cattolica del Sacro Cuore, Rome, Italy.; Grandinetti M; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Massetti M; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy; Department of Cardiovascular and Pulmonary Sciences, Università Cattolica del Sacro Cuore, Rome, Italy.; Lanza GA; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy; Department of Cardiovascular and Pulmonary Sciences, Università Cattolica del Sacro Cuore, Rome, Italy.; Limongelli G; Inherited and Rare Cardiovascular Diseases, Department of Translational Medical Sciences, University of Campania Luigi Vanvitelli, Monaldi Hospital, Naples, Italy.; Graziani F; Department of Cardiovascular Sciences, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy. Electronic address: Francesca.graziani@policlinicogemelli.it.

Publisher: Elsevier Science Country of Publication: Netherlands NLM ID: 100968751 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1872-8278 (Electronic) Linking ISSN: 15677249 NLM ISO Abbreviation: Mitochondrion Subsets: MEDLINE

Mancuso M; Department of Clinical and Experimental Medicine, Neurological Institute, University of Pisa, Pisa, Italy.; Papadopoulou MT; Department of Pediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, University Hospital of Lyon, Member of the ERN EpiCARE, Lyon, France.; Ng YS; Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute AND National Institute for Health and Care Research (NIHR) Newcastle Biomedical Research Centre (BRC), Newcastle University, Newcastle upon Tyne, UK.; NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.; Ardissone A; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Bellusci M; Reference Center for Inherited Metabolic Disorders MetabERN, Mitochondrial Disorders Research Group (imas12), '12 de Octubre' University Hospital, Madrid, Spain.; Bertini E; Research Unit of Neuromuscular Disease, Translational Pediatrics and Clinical Genetics, Bambino Gesu' Children's Research Hospital, IRCCS, Rome, Italy.; Di Vito L; IRCCS Istituto delle Scienze Neurologiche di Bologna, Full Member of the European Reference Network for Rare and Complex Epilepsies (EpiCARE), Bologna, Italy.; Evangelista T; Department of Neuropathology, Functional Unit of Neuromuscular pathology and Department of Neuromyology, Institute of Myology, EURO-NMD coordination, Pitié-Salpêtrière Hospital, APHP Sorbonne University, Paris, France.; Fons C; Epilepsy and Neurometabolics Units, Pediatric Neurology Department, Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Barcelona, Spain.; Hikmat O; Department of Paediatrics and Adolescent Medicine, Haukeland University Hospital, Bergen and Department of Clinical Medicine (K1), University of Bergen, Bergen, Norway.; Horvath R; Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.; Klopstock T; Department of Neurology, Friedrich-Baur-Institute, LMU University Hospital, Ludwig-Maximilians-Universität München, Munich, Germany.; German Center for Neurodegenerative Diseases (DZNE), Munich, Germany.; Munich Cluster for Systems Neurology (SyNergy), Munich, Germany.; Kornblum C; Department of Neurology, Section of Neuromuscular Diseases, University Hospital Bonn, Bonn, Germany.; Lamperti C; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Licchetta L; IRCCS Istituto delle Scienze Neurologiche di Bologna, Full Member of the European Reference Network for Rare and Complex Epilepsies (EpiCARE), Bologna, Italy.; Molnar MJ; Institute of Genomic Medicine and Rare Disorders, Semmelweis University, Budapest, Hungary.; Varhaug KN; Department of Neurology, Haukeland University Hospital, Bergen, Norway.; O'Callaghan M; Epilepsy and Neurometabolics Units, Pediatric Neurology Department, Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Barcelona, Spain.; Pressler RM; UCL Great Ormond Street Institute of Child Health, London, UK.; Great Ormond Street Hospital for Children, London, UK.; Schiff M; Reference Center for Mitochondrial Disorders (CARAMMEL) and Reference Center for Inborn Errors of Metabolism, Department of Pediatrics, Necker-Enfants-Malades Hospital, Assistance Publique-Hôpitaux de Paris, University of Paris-Cité, MetabERN, Paris, France.; INSERM UMRS_1163, Imagine Institute, Paris, France.; Servidei S; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Dipartimento Di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Szabo N; Saint John's Hospital, Child Epilepsy Centre, Budapest, Hungary.; Gorman GS; Wellcome Centre for Mitochondrial Research, Translational and Clinical Research Institute AND National Institute for Health and Care Research (NIHR) Newcastle Biomedical Research Centre (BRC), Newcastle University, Newcastle upon Tyne, UK.; NHS Highly Specialised Service for Rare Mitochondrial Disorders, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.; Cross JH; UCL Great Ormond Street Institute of Child Health, London, UK.; Great Ormond Street Hospital for Children, London, UK.; Rahman S; UCL Great Ormond Street Institute of Child Health, London, UK.; Great Ormond Street Hospital for Children, London, UK.

Publisher: Wiley Country of Publication: England NLM ID: 9506311 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1468-1331 (Electronic) Linking ISSN: 13515101 NLM ISO Abbreviation: Eur J Neurol Subsets: MEDLINE

Luigetti M; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168, Rome, Italy. mluigetti@gmail.com.; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy. mluigetti@gmail.com.; Vitali F; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Romano A; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168, Rome, Italy.; Sciarrone MA; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Guglielmino V; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Ardito M; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Sabino A; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Servidei S; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168, Rome, Italy.; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Piro G; Medical Oncology, Department of Medical and Surgical Sciences, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Carbone C; Medical Oncology, Department of Medical and Surgical Sciences, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Graziani F; Department of Cardiovascular Medicine, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Lillo R; Department of Cardiovascular Medicine, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy.; Department of Cardiovascular and Pneumological Sciences, Catholic University of the Sacred Heart, Rome, Italy.; Ferraro PM; Section of Nephrology, Department of Medicine, Università degli Studi di Verona, Verona, Italy.; Primiano G; Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168, Rome, Italy.; Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.; Fondazione UILDM Lazio Onlus, Rome, Italy.

Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-2322 (Electronic) Linking ISSN: 20452322 NLM ISO Abbreviation: Sci Rep Subsets: MEDLINE

Dono F; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy; Behavioral Neurology and Molecular Neurology Units, Center for Advanced Studies and Technology-CAST-, University G. D'Annunzio of Chieti-Pescara, Italy. Electronic address: fedele.dono@unich.it.; Evangelista G; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy; Behavioral Neurology and Molecular Neurology Units, Center for Advanced Studies and Technology-CAST-, University G. D'Annunzio of Chieti-Pescara, Italy.; Rodorigo D; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy.; Rollo E; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy.; Romozzi M; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy.; Corniello C; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy.; Liviello D; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy.; Dasara M; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy; Behavioral Neurology and Molecular Neurology Units, Center for Advanced Studies and Technology-CAST-, University G. D'Annunzio of Chieti-Pescara, Italy.; Capriati L; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy; Neurofisiopatologia, Dipartimento di Neuroscienze, Organi di Senso e Torace, Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Quintieri P; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy.; Servidei S; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy; Neurofisiopatologia, Dipartimento di Neuroscienze, Organi di Senso e Torace, Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address: serenella.servidei@policlinicogemelli.it.; Della Marca G; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy; Neurofisiopatologia, Dipartimento di Neuroscienze, Organi di Senso e Torace, Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address: giacomo.dellamarca@policlinicogemelli.it.; Calabresi P; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy; Neurologia, Dipartimento di Neuroscienze, Organi di Senso e Torace, Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address: paolo.calabresi@policlinicogemelli.it.; Sensi SL; Department of Neuroscience, Imaging and Clinical Sciences, 'G. d'Annunzio' University of Chieti-Pescara, Chieti, Italy; Behavioral Neurology and Molecular Neurology Units, Center for Advanced Studies and Technology-CAST-, University G. D'Annunzio of Chieti-Pescara, Italy. Electronic address: ssensi@uci.edu.; Vollono C; Dipartimento di Neuroscienze, Organi di Senso e Torace, Università Cattolica del Sacro Cuore, Rome, Italy; Neurofisiopatologia, Dipartimento di Neuroscienze, Organi di Senso e Torace, Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address: catello.vollono@policlinicogemelli.it.

Publisher: Academic Press Country of Publication: United States NLM ID: 100892858 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-5069 (Electronic) Linking ISSN: 15255050 NLM ISO Abbreviation: Epilepsy Behav Subsets: MEDLINE