Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORE (P7-9.007)

S. Finkel, Richard; Benguerba, Kamal; Gehani, Manish; Raju, Dheeraj; Faulkner, Eric; LaMarca, Nicole; Servais, Laurent

doi:10.1212/wnl.0000000000202597

Public

Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORE (P7-9.007)

Published in Neurology • Apr 25, 2023

Authors:

Richard S. Finkel

Kamal Benguerba

Manish Gehani

Abstract

Objective: We sought to describe clinical outcomes after onasemnogene abeparvovec (OA) monotherapy for patients with ≥4 SMN2 copies in RESTORE, a comprehensive, noninterventional spinal muscular atrophy (SMA) registry. Background: OA is a one-time gene replacement therapy for SMA. While clinical tri...

Subject

Milestone

Medicine

Spinal muscular atrophy

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!