Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study
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Background: LT-001 evaluated long-term safety/efficacy of onasemnogene abeparvovec (OA) for spinal muscular atrophy (SMA) patients from START (N = 15; NCT02122952). ... ...
