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Neuromuscular Disorders

Zolgensma Benefits Observed in Presymptomatic SMA Patients

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.
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