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Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen.

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Title: Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen.
Authors: Coratti G; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Bovis F; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy., Pane M; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Pasternak A; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Glanzman AM; Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Morando S; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Montes J; Columbia University Irving Medical Center, New York, New York, USA., Cavallina I; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy., Dunaway Young S; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA., Duong T; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA., Rolle E; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy., Civitello M; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., De Sanctis R; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Bravetti C; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Ricci F; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy., Gadaleta G; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Mongini T; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Pera MC; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Department of Medicine and Surgery, Child Neuropsychiatry Unit, University of Parma, Parma, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Brolatti N; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Hirano M; Columbia University Irving Medical Center, New York, New York, USA., Zolkipli-Cunningham Z; Division of Human Genetics, Children's Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Darras BT; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Bruno C; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Day J; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA., Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Finkel RS; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Mercuri E; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Corporate Authors: iSMAC/international SMA consortium
Source: European journal of neurology [Eur J Neurol] 2025 Jul; Vol. 32 (7), pp. e70268.
Publication Type: Journal Article
Journal Info: Publisher: Wiley Country of Publication: England NLM ID: 9506311 Publication Model: Print Cited Medium: Internet ISSN: 1468-1331 (Electronic) Linking ISSN: 13515101 NLM ISO Abbreviation: Eur J Neurol Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen.
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  Data: <searchLink fieldCode="AU" term="%22Coratti+G%22">Coratti G</searchLink>; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Bovis+F%22">Bovis F</searchLink>; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Pane+M%22">Pane M</searchLink>; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. 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Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Ricci+F%22">Ricci F</searchLink>; Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Gadaleta+G%22">Gadaleta G</searchLink>; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Mongini+T%22">Mongini T</searchLink>; Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy.<br /><searchLink fieldCode="AU" term="%22Sframeli+M%22">Sframeli M</searchLink>; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.<br /><searchLink fieldCode="AU" term="%22Pera+MC%22">Pera MC</searchLink>; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Department of Medicine and Surgery, Child Neuropsychiatry Unit, University of Parma, Parma, Italy.<br /><searchLink fieldCode="AU" term="%22Messina+S%22">Messina S</searchLink>; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.<br /><searchLink fieldCode="AU" term="%22D'Amico+A%22">D'Amico A</searchLink>; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Catteruccia+M%22">Catteruccia M</searchLink>; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Brolatti+N%22">Brolatti N</searchLink>; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Hirano+M%22">Hirano M</searchLink>; Columbia University Irving Medical Center, New York, New York, USA.<br /><searchLink fieldCode="AU" term="%22Zolkipli-Cunningham+Z%22">Zolkipli-Cunningham Z</searchLink>; Division of Human Genetics, Children's Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania, Philadelphia, Pennsylvania, USA.<br /><searchLink fieldCode="AU" term="%22Darras+BT%22">Darras BT</searchLink>; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Bertini+E%22">Bertini E</searchLink>; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Bruno+C%22">Bruno C</searchLink>; Center of Translational and Experimental Myology and Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Day+J%22">Day J</searchLink>; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.<br /><searchLink fieldCode="AU" term="%22Sansone+VA%22">Sansone VA</searchLink>; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Finkel+RS%22">Finkel RS</searchLink>; Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.<br /><searchLink fieldCode="AU" term="%22Mercuri+E%22">Mercuri E</searchLink>; Pediatric Neurology Unit, Catholic University of Sacred Heart, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
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