What type of study is this?

This is a Pre-Registered Trial study.

December 8, 2025Nature MedicineOpen Access

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

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Safety profile of gene transfer therapy showed adverse events consistent with treatment-experienced patients.
Fifty-five percent experienced nasopharyngitis and 51.9% experienced pyrexia, with 48.1% having treatment-related adverse events.
Multicenter study enrolled 27 spinal muscular atrophy patients, aged 2 to <18 years, who previously stopped nusinersen or risdiplam.
Findings imply a significant need for effective therapies addressing ongoing treatment challenges in spinal muscular atrophy.

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Open-label trial evaluates safety and tolerability of gene transfer therapy in spinal muscular atrophy patients previously on nusinersen.

Kwon et al. (2025) studied this question.

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