[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

^○Ling Yan¹, Nancy Kuntz², Wildon Farwell³, Zhenshao John Zhong³, Peng Sun³, Sarah Gheuens³, Eugene Schneider⁴, Richard Finkel^5,6 (Clinical Development Head, Biogen Japan¹, Division of Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA², Biogen, Cambridge, MA, USA³, Ionis Pharmaceuticals Inc., Carlsbad, CA, USA⁴, Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital, Orlando, FL, USA^{5, the ENDEAR study group6})