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Day, J.W., Finkel, R.S., Chiriboga, C.A., Connolly, A.M., Crawford, T.O., Darras, B.T., et al. (2021) Onasemnogene Abeparvovec Gene Therapy for Symptomatic Infantile-Onset Spinal Muscular Atrophy in Patients with Two Copies of SMN2 (STR1VE): An Open-Label, Single-Arm, Multicentre, Phase 3 Trial. The Lancet Neurology, 20, 284-293.
https://doi.org/10.1016/s1474-4422(21)00001-6

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