[E1] English Session 1
neurodegenerative disease / metabolic disease

[E-005] Nusinersen in infants with spinal muscular atrophy（SMA）：design／interim results of the ENDEAR study

^○Saito Kayoko¹, Nancy Kuntz², Wildon Farwell³, Zhenshao John Zhong³, Peng Sun³, Sarah Gheuens³, Eugene Schneider⁴, Richard Finkel（endear Study Group）⁵ (1.Tokyo Women’s Medical University, Institute of Medical Genetics,Tokyo, Japan, 2.Division of Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA, 3.Biogen, Cambridge, MA, USA, 4.Ionis Pharmaceuticals Inc., Carlsbad, CA, USA, 5.Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital, Orlando, FL, USA)