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
“These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.
- “These children now have an improved quality of life, vastly different from what would have been expected for them if they had not received treatment.
- Results from the IV cohort, which included 63 patients, demonstrated how a single administration of Zolgensma provided consistent, substantial and durable efficacy over time.
- “Data from the LT-001 and LT-002 studies showed that, regardless of the patient’s symptomatic status at the time of treatment, Zolgensma IV is an effective and durable treatment option.
- Nor can there be any guarantee that such products will be commercially successful in the future.