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HARVARD Citation
Mercuri, E. et al. (2021). Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet neurology. 20 (10), pp. 832-841. [Online].