AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Titel:
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Auteur:
Al-Zaidy, Samiah A. Kolb, Stephen J. Lowes, Linda Alfano, Lindsay N. Shell, Richard Church, Kathleen R. Nagendran, Sukumar Sproule, Douglas M. Feltner, Douglas E. Wells, Courtney Ogrinc, Francis Menier, Melissa L’Italien, James Arnold, W. David Kissel, John T. Kaspar, Brian K. Mendell, Jerry R.