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Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.
Pera, Maria Carmela; Coratti, Giorgia; Bovis, Francesca; Pane, Marika; Pasternak, Amy; Montes, Jacqueline; Sansone, Valeria A; Dunaway Young, Sally; Duong, Tina; Messina, Sonia; Mizzoni, Irene; D'Amico, Adele; Civitello, Matthew; Glanzman, Allan M; Bruno, Claudio; Salmin, Francesca; Morando, Simone; De Sanctis, Roberto; Sframeli, Maria; Antonaci, Laura; Frongia, Anna Lia; Rohwer, Annemarie; Scoto, Mariacristina; De Vivo, Darryl C; Darras, Basil T; Day, John; Martens, William; Patanella, Katia A; Bertini, Enrico; Muntoni, Francesco; Finkel, Richard; Mercuri, Eugenio.
Affiliation
  • Pera MC; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Coratti G; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Bovis F; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Pane M; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Pasternak A; Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.
  • Montes J; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Sansone VA; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Dunaway Young S; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Duong T; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.
  • Messina S; Departments of Rehabilitation and Regenerative Medicine and Neurology, Columbia University Irving Medical Center, New York, New York, USA.
  • Mizzoni I; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy.
  • D'Amico A; Department of Neurology, Stanford University, Palo Alto, California, USA.
  • Civitello M; Department of Neurology, Stanford University, Palo Alto, California, USA.
  • Glanzman AM; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy.
  • Bruno C; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
  • Salmin F; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
  • Morando S; Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, USA.
  • De Sanctis R; St. Jude Children's Research Hospital, Memphis, USA.
  • Sframeli M; Department of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
  • Antonaci L; Center of Experimental and Translational Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
  • Frongia AL; Neurorehabilitation Unit, University of Milan, Neuromuscular Omnicentre Clinical Center, Niguarda Hospital, Milan, Italy.
  • Rohwer A; Center of Experimental and Translational Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
  • Scoto M; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • De Vivo DC; Department of Clinical and Experimental Medicine and Centro Clinico Nemo Sud, University of Messina, Messina, Italy.
  • Darras BT; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Day J; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
  • Martens W; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy.
  • Patanella KA; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
  • Bertini E; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, United Kingdom.
  • Muntoni F; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.
  • Finkel R; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Mercuri E; Department of Neurology, Stanford University, Palo Alto, California, USA.
Ann Clin Transl Neurol ; 8(8): 1622-1634, 2021 08.
Article in En | MEDLINE | ID: mdl-34165911
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Spinal Muscular Atrophies of Childhood / Registries / Outcome Assessment, Health Care Type of study: Clinical_trials / Observational_studies Limits: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Male / Middle aged Language: En Journal: Ann Clin Transl Neurol Year: 2021 Document type: Article Affiliation country: Italia Country of publication: Estados Unidos
Fulltext
Add to My VHL
Print
XML
PubMed Links
Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Spinal Muscular Atrophies of Childhood / Registries / Outcome Assessment, Health Care Type of study: Clinical_trials / Observational_studies Limits: Adolescent / Adult / Aged / Child / Child, preschool / Female / Humans / Male / Middle aged Language: En Journal: Ann Clin Transl Neurol Year: 2021 Document type: Article Affiliation country: Italia Country of publication: Estados Unidos