[O32] 神経筋疾患 3

[O32-2] FIREFISH: 48-months efficacy and safety of risdiplam in type 1 spinal muscular atrophy

^○Yasuhiro Takedhima¹, Baranello Giovanni², Riccardo Masson³, Maria Mazurkiewicz-Beldzinska⁴, Eugenio Mercuri⁵, Laurent Servais⁶, Hui Xiong⁷, Edmar Zanoteli⁸, Muna El-Khairi⁹, Marianne Gerber⁹, Laura Palfreeman⁹, Basil T Darras¹⁰ (1.Department of Pediatrics, Hyogo College of Medicine, Japan, 2.Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health University College London, London, UK, 3.Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy, 4.Department of Developmental Neurology, Medical University of Gdansk, Gdansk, Poland, 5.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 6.MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, United Kingdom, 7.Department of Pediatrics, Peking University First Hospital, Beijing, China, 8.Department of Neurology, Faculdade de Medicina, Universidade de Sao Paulo (FMUSP), Sao Paulo, Brazil, 9.F. Hoffmann-La Roche Ltd., Basel, Switzerland, 10.Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA)