[IO4] English Session 4

[IO4-4] RAINBOWFISH: Primary efficacy and safety in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)

^○Yuh-Juh Jong¹, Richard S Finkel², Michelle A Farrar³, Laurent Servais⁴, Dmitry Vlodavets⁵, Edmar Zanoteli⁶, Mohammad Al-Muhaizea⁷, Alexandra Prufer⁸, Birgit Jaber⁹, Laura Palfreeman⁹, Eleni Gaki⁹, Michael Rabbia⁹, Enrico Bertini¹⁰ (1.Departments of Pediatrics and Laboratory Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan, 2.Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital, Memphis, USA, 3.SCHN and UNSW Medicine, UNSW Sydney, Sydney, Australia, 4.MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK, 5.Russian Children Neuromuscular Center, Russian National Research Medical University, Moscow, Russia, 6.Department of Neurology, Universidade de Sao Paulo, Sao Paulo, Brazil, 7.Department of Neurosciences, KFSH&RC, Riyadh, Saudi Arabia, 8.Pediatrics Department, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil, 9.F. Hoffmann-La Roche Ltd., Basel, Switzerland, 10.Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesu Children's Research Hospital IRCCS, Rome, Italy)