PBI1 THE VALUE OF ONASEMNOGENE ABEPARVOVEC (AVXS-101) AS A COST-EFFECTIVE GENE REPLACEMENT THERAPY FOR SPINAL MUSCULAR ATROPHY TYPE 1 IN IMPROVING SURVIVAL, HEALTHCARE RESOURCE UTILIZATION, AND MOTOR FUNCTION
Abstract
Authors
I. Jensen O. Dabbous D.C. Malone M. Droege R. Dean D.E. Feltner D.M. Sproule B. Maru R. Arjunji F. Khan