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Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy.
Pane, Marika; Coratti, Giorgia; Pera, Maria Carmela; Sansone, Valeria A; Messina, Sonia; d'Amico, Adele; Bruno, Claudio; Salmin, Francesca; Albamonte, Emilio; De Sanctis, Roberto; Sframeli, Maria; Di Bella, Vincenzo; Morando, Simone; Palermo, Concetta; Frongia, Anna Lia; Antonaci, Laura; Capasso, Anna; Catteruccia, Michela; Longo, Antonella; Ricci, Martina; Cutrona, Costanza; Pirola, Alice; Bravetti, Chiara; Pedemonte, Marina; Brolatti, Noemi; Bertini, Enrico; Mercuri, Eugenio.
  • Pane M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Coratti G; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Sansone VA; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Messina S; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
  • d'Amico A; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
  • Bruno C; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Salmin F; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.
  • Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
  • De Sanctis R; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
  • Sframeli M; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Di Bella V; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
  • Morando S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
  • Palermo C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.
  • Frongia AL; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Antonaci L; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Capasso A; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Catteruccia M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Longo A; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Ricci M; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Cutrona C; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Pirola A; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Bravetti C; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.
  • Pedemonte M; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Brolatti N; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.
  • Bertini E; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.
  • Mercuri E; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Ann Clin Transl Neurol ; 9(3): 404-409, 2022 03.
Article in English | MEDLINE | ID: covidwho-1750287
ABSTRACT
The study reports real world data in type 2 and 3 SMA patients treated for at least 2 years with nusinersen. Increase in motor function was observed after 12 months and during the second year. The magnitude of change was variable across age and functional subgroup, with the largest changes observed in young patients with higher function at baseline. When compared to natural history data, the difference between study cohort and untreated patients swas significant on both Hammersmith Functional Motor Scale and Revised Upper Limb Module both at 12 months and at 24 months.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Muscular Atrophy, Spinal Type of study: Cohort study / Observational study / Prognostic study Limits: Humans Language: English Journal: Ann Clin Transl Neurol Year: 2022 Document Type: Article Affiliation country: Acn3.51514

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Muscular Atrophy, Spinal Type of study: Cohort study / Observational study / Prognostic study Limits: Humans Language: English Journal: Ann Clin Transl Neurol Year: 2022 Document Type: Article Affiliation country: Acn3.51514