Your browser doesn't support javascript.
loading
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.
Afiliação
  • 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 em En | MEDLINE | ID: mdl-35166467
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.
Assuntos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Tipo de estudo: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Tipo de estudo: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article