書誌情報

O2-04. FIREFISH : 脊髄性筋萎縮症 (SMA) I型患者におけるリスジプラムの5年間継続投与時の有効性及び安全性

竹島泰弘1, Giovanni Baranello2, Riccardo Masson3, Maria Mazurkiewicz-Beldzinska4, Eugenio Mercuri5, Laurent Servais6, Hui Xiong7, Edmar Zanoteli8, Muna El-Khairi9, Marianne Gerber9, Laura Palfreeman9, Basil T Darras10
1兵庫医科大学小児科, 2Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 3Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, 4Department of Developmental Neurology, Medical University of Gdansk, 5Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, 6MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, 7Department of Pediatrics, Peking University First Hospital, 8Department of Neurology, Faculdade de Medicina, Universidade de Sao Paulo (FMUSP), 9F. Hoffmann - La Roche Ltd., 10Department of Neurology, Boston Children's Hospital, Harvard Medical School,
脳と発達 57(suppl): S232-S232, 2025.

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