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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
Mercuri, Eugenio; Muntoni, Francesco; Baranello, Giovanni et al.
2021 • In The Lancet Neurology, 20 (10), p. 832-841

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Onasemnogene abeparvovec gene therapy for symptomatic infantile onset spinal muscular atrophy type 1 (STR1VE-EU).pdf

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