Protocol 232SM201: An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atropy” (Protocol

Project: Research project

Project Details

Effective start/end date10/22/1510/31/25


  • Ann & Robert H. Lurie Children’s Hospital of Chicago (Prot# 232SM201 // Prot# 232SM201)
  • Biogen Inc. (Prot# 232SM201 // Prot# 232SM201)