[O32] 神経筋疾患 3

[O32-1] SUNFISH: 48-months efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy

^○Kayoko Saito¹, Tamaki Kato¹, John W Day², Nicolas Deconinck³, Elena S Mazzone⁴, Andres Nascimento⁵, Maryam Oskoui⁶, Carole Vuillerot⁷, Giovanni Baranello⁸, Laurent Servais⁹, Jessica Braid¹⁰, Marianne Gerber¹⁰, Carmen Martin¹⁰, Wai Yin Yeung¹⁰, Eugenio Mercuri⁴ (1.Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan, 2.Department of Neurology, Stanford University, Palo Alto, CA, USA, 3.Neuromuscular Reference Center, UZ Gent, Ghent, Belgium, 4.Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy, 5.Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Deu, Fundacion Sant Joan de Deu, CIBERER - ISC III, Barcelona, Spain, 6.Departments of Pediatrics and Neurology Neurosurgery, McGill University, Montreal, Canada, 7.Department of Pediatric Physical Medicine and Rehabilitation, Hopital Mere Enfant, CHU-Lyon, Bron, France, 8.Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health University College London, London, UK, 9.MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, United Kingdom, 10.F. Hoffmann-La Roche Ltd., Basel, Switzerland)