Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)
John W. Day, Claudia A. Chiriboga, Thomas O. Crawford, Basil T. Darras, Richard S. Finkel, Anne M. Connolly, Susan T. Iannaccone, Nancy L. Kuntz, Loren D.M. Peña, Perry B. Shieh, Edward C. Smith, Meredith Schultz, Douglas E. Feltner, Sitra Tauscher‐Wisniewski, Francis G. Ogrinc, Ankita Shah, Haojun Ouyang, Thomas A. Macek, Elaine Kernbauer, Douglas M. Sproule, Jerry R. Mendell
Abstract
Evaluate preliminary data from the phase 3 STR1VE (NCT03306277) study investigating efficacy and safety of one-time intravenous infusion of onasemnogene abeparvovec-xioi (formerly AVXS-101) in patients with SMA1.
Topics & Concepts
MedicineTheologyPhilosophyInternal medicineNeurogenetic and Muscular Disorders ResearchRNA Interference and Gene DeliveryRNA modifications and cancer