AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones. (June 2017)

• Record Type: Journal Article
• Authors: Mendell, Jerry R.; Al-Zaidy, Samiah; Shell, Richard; Arnold, W. David; Rodino-Klapac, Louise; Prior, Thomas W.; Lowes, Linda; Alfano, Lindsay; Berry, Katherine; Church, Kathleen; Kissel, John T.; Nagendran, Sukumar; L'Italien, James; Sproule, Douglas M.; Cardenas, Jessica; Burghes, Arthur H.M.; Foust, K.D.; Meyer, Kathrin; Likhite, Shibi; Kaspar, Brian K.
• Is Part Of: European journal of paediatric neurology. Volume 21(2017)Supplement 1
• Page Start: e13
• Page End: e14
• Publication Date: 2017-06
• Subjects: Pediatric neurology -- Periodicals; Nervous System Diseases -- Periodicals; Child -- Periodicals; Infant -- Periodicals; Neurologie pédiatrique -- Périodiques; Pediatric neurology; Electronic journals; Periodicals; Electronic journals; 618.928
• DOI: 10.1016/j.ejpn.2017.04.1216
• Languages: English
• ISSNs: 1090-3798