학술논문
AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Continued Independence from Nutritional and Ventilatory Support in Patients Dosed Early in Disease Progression
Document Type
Journal
Author
Shell, Richard; Al-Zaidy, Samiah; Arnold, W. Dave; Rodino-Klapac, Louise; Prior, Thomas; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Kotha, Kavitha; Church, Kathleen; Kissel, John; Nagendran, Sukumar; L'Italien, James; Sproule, Douglas M.; Wells, Courtney; Burghes, Arthur; Foust, Kevin D.; Meyer, Kathrin; Likhite, Shibi; Kaspar, Brian; Mendell, Jerry
Source
Subject
Language
English
ISSN
1526632X