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

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第一作者: Giorgia,Coratti
第一单位: 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.
作者单位: 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. [1] Department of Health Sciences (DISSAL), University of Genoa, Genoa, Italy. [2] Neuromuscular Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA. [3] The NEMO Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy. [4] Unit of Neuromuscular and Neurodegenerative Disorders, Translational Paediatrics and Clinical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. [5] Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. [6] 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. [7] Columbia University Irving Medical Center, New York, New York, USA. [8] Section of Child and Adolescent Neuropsychiatry, Department of Public Health and Paediatric Sciences, University of Turin, Turin, Italy. [9] Department of Neurology and Clinical Neurosciences, Stanford University, Palo Alto, California, USA. [10] Center for Experimental Neurotherapeutics, Department of Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. [11] Centro Clinico Nemo, U.O.C. Neuropsichiatria Infantile, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. [12] Neuromuscular Unit, Department of Neurosciences 'Rita Levi Montalcini', University of Turin, Turin, Italy. [13] Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy. [14] 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. [15] Division of Human Genetics, Children's Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania, Philadelphia, Pennsylvania, USA. [16]
DOI 10.1111/ene.70268
PMID 40576143
发布时间 2025-06-29
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European journal of neurology

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