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Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA.

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第一作者: Giorgia,Coratti
第一单位: Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.
作者单位: Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy. [1] St. Jude Children's Research Hospital, Memphis, TN 38105, USA. [2] Department of Developmental Neuroscience, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. [3] The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, 20162 Milan, Italy. [4] Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. [5] Columbia University Irving Medical Center, New York, NY 10032, USA. [6] Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA. [7] Departments of Neurology TD, Stanford University, Palo Alto, CA 94305, USA. [8] Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Medical Genetics, Bambino Gesù Children's Hospital, IRCCS, 00165 Rome, Italy. [9] Department of Clinical and Experimental Medicine, University of Messina, 98122 Messina, Italy. [10] 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, 16132 Genoa, Italy. [11] Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; University of Pennsylvania, Philadelphia, PA 19104, USA. [12] Department of Neurology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK; School of Health and Sports Sciences, University of Suffolk, Ipswich IP4 1QJ, UK. [13] Pediatric Neurology Unit, Catholic University, 00135 Rome, Italy; Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy. Electronic address: eugeniomaria.mercuri@unicatt.it. [14]
DOI 10.1016/j.nmd.2024.05.003
PMID 38936290
发布时间 2025-07-19
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Neuromuscular disorders : NMD

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