1. B.01 AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update. (5th June 2019) Authors: Day, JW; Chiriboga, CA; Crawford, TO; Darras, BT; Finkel, RS; Connolly, AM; Iannaccone, ST; Kuntz, NL; Pena, LD; Schultz, M; Shieh, PB; Smith, EC; Feltner, DE; Ogrinc, FG; Macek, TA; Kernbauer, E; Muehring, LM; L'Italien, J; Sproule, DM; Kaspar, BK Journal: Canadian journal of neurological sciences Issue: Volume 46(2019)Supplement 1 Page Start: S10 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. B.01 AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update. (June 2019) Authors: Day, JW; Chiriboga, CA; Crawford, TO; Darras, BT; Finkel, RS; Connolly, AM; Iannaccone, ST; Kuntz, NL; Pena, LD; Schultz, M; Shieh, PB; Smith, EC; Feltner, DE; Ogrinc, FG; Macek, TA; Kernbauer, E; Muehring, LM; L'Italien, J; Sproule, DM; Kaspar, BK Journal: Canadian journal of neurological sciences Issue: Volume 46(2019)Supplement 1 Page Start: S10 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗