Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2022
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.
Epistemonikos ID: 28a11ea4d06b3f5c529cdde8296b7b214b9b24fb
First added on: May 09, 2024