Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1
O. Dabbous, B. Maru, J. Jansen, M. Lorenzi, M. Cloutier, A. Guerin, I. Pivneva, E. Wu, R. Arjunji, D. Feltner, D. Sproule
Advances in Therapy