1. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones. (June 2017) 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.; F... Journal: European journal of paediatric neurology Issue: Volume 21(2017)Supplement 1 Page Start: e13 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗