Abstract
To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment
naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age
over a 15 month trial duration.
Stanley Manne Children's Research Institute
Louis A. Simpson and Kimberly K. Querrey Biomedical Research Center
303 East Superior Street
Chicago, IL 60611
