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

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Journal

The Lancet Neurology

Topics

Medicine

Internal Medicine

Pediatrics

Surgery

Genetics

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DOI

10.1016/s1474-4422(21)00001-6