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Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Muscular Atrophy (ISIS 396443-CS3B)
This study is to assess the efficacy and safety of multiple doses of the study medication, ISIS 396443, given intrathecally to subjects with infantile-onset SMA.