P223 Long-term follow-up of onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy (SMA) type 1

In the phase 1 trial, START, SMA type 1 patients who received the proposed therapeutic dose of onasemnogene abeparvovec (n=12) demonstrated substantially improved outcomes versus natural history. We evaluated long-term safety and efficacy of onasemnogene abeparvovec for patients with SMA type 1 (biallelic SMN1 mutations/deletions; two SMN2 copies) enrolled in the LT-001 study. The primary objective was to evaluate long-term safety assessed by medical history and record review, physical examination, laboratory evaluation, and pulmonary assessments.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: J. Mendell, M. Wigderson, I. Alecu, L. Yang, L. Mehl, A. Connolly Source Type: research