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Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies.
Pane, Marika; Berti, Beatrice; Capasso, Anna; Coratti, Giorgia; Varone, Antonio; D'Amico, Adele; Messina, Sonia; Masson, Riccardo; Sansone, Valeria Ada; Donati, Maria Alice; Agosto, Caterina; Bruno, Claudio; Ricci, Federica; Pini, Antonella; Gagliardi, Delio; Filosto, Massimiliano; Corti, Stefania; Leone, Daniela; Palermo, Concetta; Onesimo, Roberta; De Sanctis, Roberto; Ricci, Martina; Bitetti, Ilaria; Sframeli, Maria; Dosi, Claudia; Albamonte, Emilio; Ticci, Chiara; Brolatti, Noemi; Bertini, Enrico; Finkel, Richard; Mercuri, Eugenio.
Afiliación
  • Pane M; Paediatric Neurology, Catholic University, Rome, Italy.
  • Berti B; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Capasso A; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Coratti G; Paediatric Neurology, Catholic University, Rome, Italy.
  • Varone A; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • D'Amico A; Paediatric Neurology, Catholic University, Rome, Italy.
  • Messina S; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Masson R; Department of Neurosciences, Paediatric Neurology, Santobono-Pausilipon Children's Hospital, Naples, Italy.
  • Sansone VA; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.
  • Donati MA; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
  • Agosto C; Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, Italy.
  • Bruno C; Neurorehabilitation Unit, Centro Clinico Nemo, Niguarda Hospital, University of Milan, Milano, Italy.
  • Ricci F; Metabolic and Muscular Unit, Meyer Children's Hospital IRCCS, Florence, Italy.
  • Pini A; Dipartimento di Salute della Donna e del Bambino, Università di Padova, Padua, Italy.
  • Gagliardi D; Center of Myology and Neurodegenerative Disorders, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
  • Filosto M; AOU Città della Salute e della Scienza di Torino, Presidio OIRM (SC Neuropsichiatria Infantile), Turin, Italy.
  • Corti S; Neuromuscular Pediatric Unit, IRCCS Istituto delle Scienze Neurologiche di Bologna-UOC Neuropsichiatria dell'Età Pediatrica, Bologna, Italy.
  • Leone D; Pediatric Neurology Unit, Pediatric Hospital "Giovanni XXIII", Bari, Italy.
  • Palermo C; Department of Clinical and Experimental Sciences, NeMO-Brescia Clinical Center for Neuromuscular Diseases, University of Brescia; Brescia, Italy.
  • Onesimo R; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Milan, Italy.
  • De Sanctis R; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Ricci M; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Bitetti I; Rare Disease Unit, Pediatric Unit - Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Sframeli M; Paediatric Neurology, Catholic University, Rome, Italy.
  • Dosi C; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Albamonte E; Paediatric Neurology, Catholic University, Rome, Italy.
  • Ticci C; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Brolatti N; Department of Neurosciences, Paediatric Neurology, Santobono-Pausilipon Children's Hospital, Naples, Italy.
  • Bertini E; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.
  • Finkel R; Fondazione IRCCS Istituto Neurologico Carlo Besta Developmental Neurology Unit, Milan, Italy.
  • Mercuri E; Neurorehabilitation Unit, Centro Clinico Nemo, Niguarda Hospital, University of Milan, Milano, Italy.
EClinicalMedicine ; 59: 101997, 2023 May.
Article en En | MEDLINE | ID: mdl-37197706
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies / Risk_factors_studies Idioma: En Revista: EClinicalMedicine Año: 2023 Tipo del documento: Article País de afiliación: Italia
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies / Risk_factors_studies Idioma: En Revista: EClinicalMedicine Año: 2023 Tipo del documento: Article País de afiliación: Italia