Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.

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Title: Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.
Authors: Coratti G; Pediatric Neurology Unit, Catholic University, 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., Pera MC; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Civitello M; St Jude Children's Research Hospital, Memphis, Tennessee, USA., Rohwer A; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Salmin F; NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Glanzman AM; Department of Physical Therapy Rehabilitation, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA., Montes J; Columbia University Irving Medical Center, New York, New York, USA., Pasternak A; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., De Sanctis R; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, 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., Mizzoni I; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, Rome, Italy., Milev E; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Sframeli M; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Morando S; Center of Translational and Experimental Myology, Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Albamonte E; NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, Rome, Italy., Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, Rome, Italy., Brolatti N; Center of Translational and Experimental Myology, Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Pane M; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Scoto M; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Escudero JE; Neuromuscular Unit, Department of Neurology, Hospital Sant Joan de Déu, CIBERER - ISCIII, Barcelona, Spain.; Applied Research in Neuromuscular Diseases, Institut de Recerca Sant Joan de Déu, Barcelona, Spain., De Waele L; Department of Child Neurology, University Hospital Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium., Hirano M; Columbia University Irving Medical Center, New York, New York, USA., Zolkipli-Cunningham Z; Division of Human Genetics, Department of Pediatrics, Children's Hospital of Philadelphia, 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, Rome, Italy., Nascimiento Osorio A; Neuromuscular Unit, Department of Neurology, Hospital Sant Joan de Déu, CIBERER - ISCIII, Barcelona, Spain.; Applied Research in Neuromuscular Diseases, Institut de Recerca Sant Joan de Déu, Barcelona, Spain., Bruno C; Center of Translational and Experimental Myology, Department of Neuroscience, Rehabilitation, Ophthalmology Genetics, Maternal and Child Health, IRCCS Istituto Giannina Gaslini and University of Genoa, Genoa, Italy., Goemans N; Department of Child Neurology, University Hospital Leuven, Leuven, Belgium.; Department of Development and Regeneration, KU Leuven, Leuven, Belgium., Sansone VA; NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Day J; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA., Baranello G; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Muntoni F; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK., Finkel R; Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy., Mercuri E; Pediatric Neurology Unit, Catholic University, 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] 2024 Dec; Vol. 31 (12), pp. e16517. Date of Electronic Publication: 2024 Oct 11.
Publication Type: Journal Article; Multicenter Study
Journal Info: Publisher: Wiley Country of Publication: England NLM ID: 9506311 Publication Model: Print-Electronic 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: Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.
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  Data: <searchLink fieldCode="AU" term="%22Coratti+G%22">Coratti G</searchLink>; Pediatric Neurology Unit, Catholic University, 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="%22Pera+MC%22">Pera MC</searchLink>; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Civitello+M%22">Civitello M</searchLink>; St Jude Children's Research Hospital, Memphis, Tennessee, USA.<br /><searchLink fieldCode="AU" term="%22Rohwer+A%22">Rohwer A</searchLink>; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Salmin+F%22">Salmin F</searchLink>; NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Glanzman+AM%22">Glanzman AM</searchLink>; Department of Physical Therapy Rehabilitation, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.<br /><searchLink fieldCode="AU" term="%22Montes+J%22">Montes J</searchLink>; Columbia University Irving Medical Center, New York, New York, USA.<br /><searchLink fieldCode="AU" term="%22Pasternak+A%22">Pasternak A</searchLink>; Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22De+Sanctis+R%22">De Sanctis R</searchLink>; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Dunaway+Young+S%22">Dunaway Young S</searchLink>; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.<br /><searchLink fieldCode="AU" term="%22Duong+T%22">Duong T</searchLink>; Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA.<br /><searchLink fieldCode="AU" term="%22Mizzoni+I%22">Mizzoni I</searchLink>; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Milev+E%22">Milev E</searchLink>; Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Neuromuscular Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.<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="%22Morando+S%22">Morando S</searchLink>; Center of Translational and Experimental Myology, 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="%22Albamonte+E%22">Albamonte E</searchLink>; NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, 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, 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, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Brolatti+N%22">Brolatti N</searchLink>; Center of Translational and Experimental Myology, 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="%22Pane+M%22">Pane M</searchLink>; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. 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Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
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