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.

Principal Investigator

CONTACT INFORMATION

Primary Contact:
Shirley Paulose
(650) 724-3792