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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

Rare DiseasesSpinal Cord InjuryMedicineDegenerative DiseaseNeurologyNeuromuscular PathologyNeuromusculoskeletal DisorderPhase 3