The P-III (SPR1NT) trial evaluates Zolgensma in patients with SMA & 2 or 3 copies of SMN2. The trial reinforces the transformational benefit of early treatment with one-time gene therapy
The results showed that all children treated symptomatically in both copy cohorts met the 1EPs of sitting alone for ≥30 sec. & standing alone for ≥3 sec. with 79% & 93% achieved milestones within the WHO window of normal development. In the two-copy cohort, patients were able to stand & walk independently
In the three-copy cohort, 93% were walked alone with 73% within the WHO window of normal development, AEs were consistent with prior data with no new safety signals, and all children were free of respiratory, nutritional support & serious TRAEs
Ref: Novartis| Image:Novartis
