Type I spinal muscular atrophy patients treated with nusinersen: 4-year follow-up of motor, respiratory and bulbar function.

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第一作者： Marika,Pane

第一单位： Paediatric Neurology, Catholic University, Rome, Italy.

作者： Marika,Pane ^[1] ; Giorgia,Coratti ^[2] ; Valeria A,Sansone ^[1] ; Sonia,Messina ^[2] ; Michela,Catteruccia ^[3] ; Claudio,Bruno ^[4] ; Maria,Sframeli ^[5] ; Emilio,Albamonte ^[6] ; Marina,Pedemonte ^[4] ; Noemi,Brolatti ^[3] ; Irene,Mizzoni ^[6] ; Adele,D'Amico ^[6] ; Chiara,Bravetti ^[5] ; Beatrice,Berti ^[5] ; Concetta,Palermo ^[2] ; Daniela,Leone ^[2] ; Francesca,Salmin ^[2] ; Roberto,De Sanctis ^[2] ; Maria Carmela,Pera ^[3] ; Marco,Piastra ^[2] ; Orazio,Genovese ^[1] ; Federica,Ricci ^[2] ; Ilaria,Cavallina ^[7] ; Riccardo,Masson ^[7] ; Riccardo,Zanin ^[8] ; Caterina,Agosto ^[8] ; Eleonora,Salomon ^[9] ; Irene,Bruno ^[9] ; Andrea,Magnolato ^[10] ; Enrico,Bertini ^[10] ; Francesco Danilo,Tiziano ^[11] ; Francesca,Bovis ^[11] ; Eugenio,Mercuri ^[5] ; Italian EAP Working Group ^[12]

作者单位： Paediatric Neurology, Catholic University, Rome, Italy. ^[1] Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy. ^[2] Neurorehabilitation Unit, University of Milan, Milan, Italy. ^[3] Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy. ^[4] Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy. ^[5] Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy. ^[6] Pediatric Intensive Care Unit, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy. ^[7] AOU Città della Salute e della Scienza di Torino, Presidio OIRM (SC Neuropsichiatria Infantile), Turin, Italy. ^[8] Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, Italy. ^[9] Dipartimento di Salute della Donna e del Bambino, Università di Padova, Padua, Italy. ^[10] Institute for Maternal and Child Health, IRCCS, Trieste, Italy. ^[11] Institute of Genomic Medicine, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy. ^[12] Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy. ^[13]

关键词 long-term results longitudinal study motor function nusinersen spinal muscular atrophy