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Nusinersen in type 1 spinal muscular atrophy: Twelve-month real-world data.
Pane, Marika; Coratti, Giorgia; Sansone, Valeria A; Messina, Sonia; Bruno, Claudio; Catteruccia, Michela; Sframeli, Maria; Albamonte, Emilio; Pedemonte, Marina; D'Amico, Adele; Bravetti, Chiara; Berti, Beatrice; Brigati, Giorgia; Tacchetti, Paola; Salmin, Francesca; de Sanctis, Roberto; Lucibello, Simona; Piastra, Marco; Genovese, Orazio; Bertini, Enrico; Vita, Giuseppe; Tiziano, Francesco Danilo; Mercuri, Eugenio.
Afiliação
  • Pane M; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Coratti G; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Sansone VA; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan.
  • Messina S; Department of Clinical and Experimental Medicine, University of Messina and Neuromuscular Omnicentre Clinical Center, Messina.
  • Bruno C; Center of Myology and Neurodegenerative Disorders, Giannina Gaslini Institute, Genoa.
  • Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Baby Jesus Children's Hospital, Rome.
  • Sframeli M; Department of Clinical and Experimental Medicine, University of Messina and Neuromuscular Omnicentre Clinical Center, Messina.
  • Albamonte E; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan.
  • Pedemonte M; Center of Myology and Neurodegenerative Disorders, Giannina Gaslini Institute, Genoa.
  • D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Baby Jesus Children's Hospital, Rome.
  • Bravetti C; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Berti B; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Brigati G; Center of Myology and Neurodegenerative Disorders, Giannina Gaslini Institute, Genoa.
  • Tacchetti P; Center of Myology and Neurodegenerative Disorders, Giannina Gaslini Institute, Genoa.
  • Salmin F; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan.
  • de Sanctis R; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Lucibello S; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Piastra M; Pediatric Intensive Care Unit, Catholic University and Gemelli General Hospital, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Genovese O; Pediatric Intensive Care Unit, Catholic University and Gemelli General Hospital, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Baby Jesus Children's Hospital, Rome.
  • Vita G; Department of Clinical and Experimental Medicine, University of Messina and Neuromuscular Omnicentre Clinical Center, Messina.
  • Tiziano FD; Institute of Genomic Medicine, Catholic University and Gemelli General Hospital, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
  • Mercuri E; Paediatric Neurology and Neuromuscular Omnicentre Clinical Center, Agostino Gemelli University Polyclinic Foundation, Scientific Institute for Research and Health Care, Rome.
Ann Neurol ; 86(3): 443-451, 2019 09.
Article em En | MEDLINE | ID: mdl-31228281

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofias Musculares Espinais da Infância Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Ann Neurol Ano de publicação: 2019 Tipo de documento: Article País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofias Musculares Espinais da Infância Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Ann Neurol Ano de publicação: 2019 Tipo de documento: Article País de publicação: Estados Unidos