JSCN2019

Presentation information

English session

Nerve / Muscular disease

[E4] English session
Nerve / Muscular disease

Fri. May 31, 2019 3:30 PM - 4:30 PM Room 8 (Conference Room 131+132, 3F, Bldg. 1)

Chair: Yuka Ishikawa (Department of Pediatrics, Yakumo National Hospital), Yasuhiro Takeshima (Department of Pediatrics, Hyogo College of Medicine)

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

Laurent Servais1,2, Giovanni Baranello3, John W. Day4, Nicolas Deconinck5, Eugenio Mercuri6, Andrea Klein7, Basil Darras8, Riccardo Masson1,2, 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)

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