Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Title:
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Author:
Lowes, Linda P. Alfano, Lindsay N. Arnold, W. David Shell, Richard Prior, Thomas W. McColly, Markus Lehman, Kelly J. Church, Kathleen Sproule, Douglas M. Nagendran, Sukumar Menier, Melissa Feltner, Douglas E. Wells, Courtney Kissel, John T. Al-Zaidy, Samiah Mendell, Jerry