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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ISSN:1468-1331
DOI:10.1111/ene.16517