Presentation Information
[O2-04]FIREFISH Parts 1 and 2: 5-year efficacy and safety of risdiplam in Type I SMA
○Yasuhiro Takeshima1, Giovanni Baranello2, 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, 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)