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Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany.

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第一作者： Astrid,Pechmann

第一单位： Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Germany.

作者： Astrid,Pechmann ^[1] ; Thorsten,Langer ^[1] ; David,Schorling ^[1] ; Sabine,Stein ^[1] ; Sibylle,Vogt ^[1] ; Ulrike,Schara ^[2] ; Heike,Kölbel ^[2] ; Oliver,Schwartz ^[3] ; Andreas,Hahn ^[4] ; Kerstin,Giese ^[4] ; Jessika,Johannsen ^[5] ; Jonas,Denecke ^[5] ; Claudia,Weiß ^[6] ; Manuela,Theophil ^[7] ; Janbernd,Kirschner ^[1]

作者单位： Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Faculty of Medicine, University of Freiburg, Germany. ^[1] Department of Neuropediatrics, Children's Hospital 1, University of Duisburg-Essen, Essen, Germany. ^[2] Department of Neuropediatrics, University Children's Hospital Muenster, Muenster, Germany. ^[3] Departement of Child Neurology, University Hospital Giessen, Justus-Liebig-University, Giessen, Germany. ^[4] Department of Pediatrics, Neuropediatrics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. ^[5] Department of Neuropediatrics, Charité - Universitätsmedizin Berlin, Berlin, Germany. ^[6] Department of Pediatrics and Neuropediatrics, DRK Klinikum Westend, Berlin, Germany. ^[7]

关键词 SMArtCARE Spinal muscular atrophy motor function motor milestones nusinersen