[O-46] Oral Session 46 Clinical trial and Regenerative medicine3

[O-46-6] FIREFISH: 24-month efficacy and safety of risdiplam in infants with Type 1 spinal muscular atrophy

^○Yasuhiro Takeshima¹, 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.Department of Pediatrics, Hyogo College of Medicine, Hyogo, Japan, 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)