AVXS-101 Gene Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update


Conference Paper

Full Text

Duke Authors

Cited Authors

  • Shell, R; Day, J; Chiriboga, C; Crawford, TO; Darras, BT; Finkel, R; Connolly, AM; Iannaccone, ST; Kuntz, NL; Pena, LDM; Schultz, M; Shieh, PB; Smith, EC; Feltner, DE; Ogrinc, FG; Macek, TA; Wells, C; Muehring, LM; L'Italien, J; Sproule, DM; Kaspar, BK; Mendell, JR

Published Date

  • January 1, 2019

Published In

Volume / Issue

  • 199 /

Published By


  • 2

Electronic International Standard Serial Number (EISSN)

  • 1535-4970

International Standard Serial Number (ISSN)

  • 1073-449X

Conference Name

  • International Conference of the American-Thoracic-Society

Conference Location

  • Dallas, TX

Conference Start Date

  • May 17, 2019

Conference End Date

  • May 22, 2019