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Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.

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Title: Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
Authors: Wolfe A; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; Evelina London Children's Hospital, Guys and St Thomas' NHS Foundation Trust, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Stimpson G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Ramsey D; University of Suffolk, Ipswich, UK., 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., Dunaway Young S; Stanford University, Palo Alto, USA., Mayhew A; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Integrated Laboratory Medicine, Newcastle Upon Tyne Hospitals NHS Foundation Trust, UK., 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., Rohwer A; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Muni Lofra R; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Integrated Laboratory Medicine, Newcastle Upon Tyne Hospitals NHS Foundation Trust, UK., Duong T; Stanford University, Palo Alto, USA., O'Reilly E; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Milev E; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Civitello M; St. Jude Children's Research Hospital, Memphis, USA.; Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, USA., Sansone VA; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, 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., Albamonte E; The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy., Mazzone E; Pediatric Neurology Unit, Catholic University, Rome, Italy.; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Main M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK., Montes J; Columbia University Irving Medical Center, New York, USA., Glanzman AM; Children's Hospital of Philadelphia, Philadelphia, USA., Zolkipli-Cunningham Z; Children's Hospital of Philadelphia, Philadelphia, USA.; Department of Pediatrics, University of Pennsylvania, PA, USA., Pasternak A; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Marini-Bettolo C; John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University, Integrated Laboratory Medicine, Newcastle Upon Tyne Hospitals NHS Foundation Trust, UK., Day JW; Stanford University, Palo Alto, USA., Darras BT; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., De Vivo DC; Columbia University Irving Medical Center, New York, USA., Baranello G; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Scoto M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK., Finkel RS; St. Jude Children's Research Hospital, Memphis, USA.; Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, USA., 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., Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK.
Corporate Authors: international SMA consortium (iSMAc)
Source: Journal of neuromuscular diseases [J Neuromuscul Dis] 2024; Vol. 11 (3), pp. 665-677.
Publication Type: Journal Article
Journal Info: Publisher: SAGE Publications Country of Publication: United States NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:2214-3602
DOI:10.3233/JND-230211