[E4] English session
Nerve / Muscular disease

[E-022] AVXS-101 Gene Replacement for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Study (STR1VE) Update

John W. Day¹, Claudia Chiriboga², Thomas O. Crawford³, Richard S. Finkel⁴, Kayoko Saito⁵, Mari Matsuo⁵, Tamaki Kato⁵, Authorship Truncated⁶, Douglas M. Sproule⁷, Jerry R. Mendell⁸ (1.Stanford University Medical Center, Stanford, CA, USA, 2.Columbia University Medical Center, New York, NY, USA, 3.Johns Hopkins Medicine, Baltimore, MD, USA, 4.Nemours Childrens Hospital, Orlando, FL, USA, 5.Tokyo Womens Medical University, Tokyo, Japan, 6.Complete author list will be included at presentation, 7.AveXis, Inc., Bannockburn, IL, USA, 8.Center for Gene Therapy Nationwide Childrens Hospital, Columbus, OH, USA)