Key result
In 168 patients with spinal muscular atrophy treated with onasemnogene abeparvovec monotherapy, 100% maintained or achieved motor milestones and 48.5% experienced an adverse event.
Why the study?
Long-term, real-world effectiveness and safety data of disease-modifying treatments for spinal muscular atrophy are needed for a larger number and broader range of patients than included in clinical trials.
Does onasemnogene abeparvovec monotherapy improve motor milestones and maintain safety in patients with spinal muscular atrophy in a real-world setting?
Population
168 patients with SMA treated with onasemnogene abeparvovec monotherapy
Design
Prospective multicenter multinational observational registry
Authors
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Real-world SMA outcomes with onasemnogene abeparvovec align with trials; extends evidence to broader populations but remains hypothesis-generating pending RCTs.
Observational (n=168)
Yes
Does onasemnogene abeparvovec monotherapy improve motor milestones and maintain safety in patients with spinal muscular atrophy in a real-world setting?
Real-world data from the RESTORE registry confirms the effectiveness and safety of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy, with better outcomes observed in those identified by newborn screening.
Servais et al. (2024) conducted an observational in Spinal muscular atrophy (SMA) (n=168). Onasemnogene abeparvovec monotherapy was evaluated on Maintenance or achievement of motor milestones. In 168 patients with spinal muscular atrophy treated with onasemnogene abeparvovec monotherapy, 100% maintained or achieved motor milestones and 48.5% experienced an adverse event.
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