AVXS-101 gene replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal studies clinical update (STR1VE-EU and STR1VE)


Conference Paper

Full Text

Duke Authors

Cited Authors

  • Mercuri, E; Baranello, G; Day, JW; Bruno, C; Corti, S; Chiriboga, CA; Crawford, TO; Darras, BT; Finkel, RS; Connolly, AM; Iannaccone, ST; Kuntz, NL; Masson, R; Pena, LD; Schultz, M; Shieh, PB; Smith, EC; Feltner, DE; Ouyang, H; Lavrov, A; Truncated, A; L'italien, J; Sproule, DM; Mendell, JR; Muntoni, F

Published Date

  • July 1, 2019

Published In

Volume / Issue

  • 26 /

Start / End Page

  • 224 - 225

Published By


  • 2

Electronic International Standard Serial Number (EISSN)

  • 1468-1331

International Standard Serial Number (ISSN)

  • 1351-5101

Conference Name

  • 5th Congress of the European-Academy-of-Neurology (EAN)

Conference Location

  • Oslo, NORWAY

Conference Start Date

  • June 29, 2019

Conference End Date

  • July 2, 2019