Safety and efficacy of intravenous onasemnogene abeparvovec in patients with spinal muscular atrophy: interim findings from the phase 3 SMART study

H. McMillan, G. Baranello,M. Farrar, C. Zaidman, J. Seibert, R. Bernardo, I. Alecu, F. Freischlager, F. Muntoni

NEUROMUSCULAR DISORDERS(2023)

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intravenous onasemnogene abeparvovec,spinal muscular atrophy
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