Concepedia

Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)

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,

Neurology · 2020 · 19 citations · 0 references

DOI

Concepts

MedicineOncologyPharmacotherapyGene TherapyImmunotherapyMolecular OncologyNeurological Gene TherapySafety Of Gene TherapySpinal Muscular AtrophyOnasemnogene Abeparvovec TherapySpinal Muscular Atrophy Pharmacotherapy

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.