[E4] English Session 4
神経・筋疾患

[E-020] FIREFISH Part 1：1-year motor function results in infants with Type 1 spinal muscular atrophy（SMA）

Laurent Servais^1,2, Giovanni Baranello³, John W. Day⁴, Nicolas Deconinck⁵, Eugenio Mercuri⁶, Andrea Klein⁷, Basil Darras⁸, Riccardo Masson^1,2, Heidemarie Kletzl⁹, Muna El-Khairi¹⁰, Timothy Seabrook⁹, Marianne Gerber⁹, Ksenija Gorni⁹, Omar Khwaja⁹ (1.Institute of Myology, Paris, France, 2.Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium, 3.Carlo Besta Neurological Research Institute Foundation, Developmental Neurology Unit, Milan, Italy, 4.Department of Neurology, Stanford University, Palo Alto, CA, USA, 5.Queen Fabiola Children’s University Hospital, Université Libre de Bruxelles, Brussels, Belgium; Neuromuscular Reference Center UZ Ghent; Ghent, Belgium, 6.Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy, 7.University Children’s Hospital Basel, Basel, Switzerland; Inselspital, Bern, Switzerland, 8.Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, 9.Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland, 10.Roche Products Ltd, Welwyn Garden City, UK)