PSY16 - HEALTH OUTCOME IMPROVEMENTS IN SPINAL MUSCULAR ATROPHY TYPE 1 PATIENTS WITH AVXS-101 GENE REPLACEMENT THERAPY
Abstract
Authors
S. Al-Zaidy A.S. Pickard K. Kotha L. Alfano L. Lowes G. Paul K. Church K. Lehman D.M. Sproule O. Dabbous B. Maru K. Berry W.D. Arnold J.T. Kissel J.R. Mendell R. Shell