Novartis Reports Long-Term Outcomes for Zolgensma Gene Therapy in SMA

Novartis today presented long-term follow-up data for Zolgensma (onasemnogene abeparvovec) in patients with spinal muscular atrophy (SMA). The data showed that patients who received Zolgensma as infants maintained significant motor function improvements over multiple years of follow-up. Patients who received treatment before symptom onset showed the best long-term outcomes, highlighting the importance of early intervention.