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The phase 3 SPR1NT trial is an ongoing open-label, single-arm study evaluating onasemnogene in presymptomatic patients (≤6 weeks of age) with a genetic diagnosis of SMA and 2 or 3 copies of survival motor neuron 2 gene.

The system is currently being assessed for its use in stroke rehabilition in the Phase 3 supplemental study ‘E-FIT’, involving 60 patients.