Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.

Nat Med · 2026 · PMC12920101 · PMID 41360993

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a positive trendno p-value reported

In both age subgroups, there was a positive trend consistently favoring the OAV101 IT group compared with sham.

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