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

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.<b>Trial registration</b>: Clinicaltrials.gov NCT identifier NCT04174157, registration date September 12, 2019.

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Publication Details

Journal
Journal of Neuromuscular Diseases
Published
2025-11-11
DOI
https://doi.org/10.1177/22143602251395173
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
Field-Weighted Citation Impact
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article

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

Dheeraj Raju, Yasemin Erbaş, Ryosuke Bo, Natalie L. Goedeker et al.
Journal of Neuromuscular Diseases
Neurogenetic and Muscular Disorders Research
article

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

Dheeraj Raju, Yasemin Erbaş, Ryosuke Bo, Natalie L. Goedeker, Laurent Servais, Sandra P. Reyna, Megan A. Waldrop, Kamal Benguerba, Perry B. Shieh, Richard S. Finkel, David Wolff
article en

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.<b>Trial registration</b>: Clinicaltrials.gov NCT identifier NCT04174157, registration date September 12, 2019.

Journal of Neuromuscular Diseases
Novartis (Switzerland) (CH), St. Jude Children's Research Hospital (US), Nationwide Children's Hospital (US), University of Liège (BE), University of California, Los Angeles (US), Tilburg University (NL), Washington University in St. Louis (US), University of Oxford (GB), Oxford Biomedical Research (US), Rode Kruis-Vlaanderen (BE), Oxford BioMedica (United Kingdom) (GB), The Ohio State University (US), Kobe University (JP)
Openalex Percentile: Top 53%
Neurogenetic and Muscular Disorders Research
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