Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry

Publication details

Published 2025
Spinal Muscular Atrophy (SMA)
University of Oxford
Erbaş Y, Servais L, Shieh PB, Goedeker NL, Waldrop MA, Bo R, Raju D, Benguerba K, Reyna SP, Wolff D, Finkel RS
DOI: 10.1177/22143602251395173. Online ahead of print.
Citation: J Neuromuscul Dis. 2025 Nov 11:22143602251395173.

Abstract

We evaluated the safety and effectiveness of onasemnogene abeparvovec (OA) for 34 patients with spinal muscular atrophy (SMA) and tracheostomies enrolled in the RESTORE registry. Most patients (76.5%) received other SMA treatments before and/or after OA. Safety findings were consistent with OA's established profile. There were four fatalities, three caused by respiratory events unrelated to OA, one of unknown cause. Overall, patients exhibited positive motor outcomes, even in cases where the need for tracheostomy emerged after treatment. These real-world data support OA treatment for patients with SMA and tracheostomies and can inform future access, treatment, and care decisions.Trial registration: Clinicaltrials.gov NCT identifier NCT04174157, registration date September 12, 2019.

Keywords: onasemnogene abeparvovec; registry; respiratory; spinal muscular atrophy; tracheostomy; ventilation support.