CM4 TIME TO TREATMENT EFFECT, EVENT-FREE SURVIVAL, AND MOTOR MILESTONE ACHIEVEMENT IN TYPE I SPINAL MUSCULAR ATROPHY PATIENTS TREATED WITH ONASEMNOGENE ABEPARVOVEC (AVXS-101) OR NUSINERSEN CONTRASTED TO NATURAL HISTORY
Abstract
Authors
D.M. Sproule B. Maru O. Dabbous R. Arjunji F. Khan D.E. Feltner M. Menier F.G. Ogrinc A. Novack M. Droege