Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset
Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion
All children (100%) in the presymptomatic intravenous cohort of LT-002 maintained or achieved all assessed motor milestones, including independent walking
To date, more than 3,000 children with spinal muscular atrophy have been treated with Zolgensma across clinical trials, managed access programs and in the commercial setting1
Additionally, children with SMA Type 2 treated with investigational intrathecal OAV101 maintained or achieved new development gains
Basel, March 20, 2023 — Novartis today presented new data which underscore the transformational and sustained benefit of Zolgensma® (onasemnogene abeparvovec), an essential one-time gene therapy for the treatment of spinal muscular...
