[O-46] 一般演題口演セッション46 治療創薬、再生医療3

[O-46-6] FIREFISH：脊髄性筋萎縮症I型患者におけるリスジプラム24ヵ月投与時の有効性・安全性

^○竹島 泰弘¹, Basil T Darras^2,5,8, Riccardo Masson³, Maria Mazurkiewicz-beldzinska⁴, Kristy Rose⁵, Hui Xiong⁶, Edmar Zanoteli⁷, Angela Dodman⁸, Muna El-Khairi⁸, Marianne Gerber⁸, Ksenija Gorni⁸, Heidemarie Kletzl⁸, Renata S Scalco⁸, Laurent Servais⁹ (1.兵庫医科大学病院 小児科, 2.Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, 3.Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy, 4.Department of Developmental Neurology Medical University of Gdansk, Gdansk, Poland, 5.Paediatric Gait Analysis Service of New South Wales, The Children’s Hospital at Westmead, Sydney, Australia, 6.Department of Pediatrics, Peking University First Hospital, Beijing, China, 7.Department of Neurology, Faculdade de Medicina, Universidade de São Paulo (FMUSP), São Paulo, Brazil, 8.F. Hoffmann-La Roche Ltd, Basel, Switzerland, 9.MDUK Oxford Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, UK)