Shots:

• The two-copy cohort of P-III SPR1NT study involves evaluating safety & efficacy of Zolgensma (one-time IV infusion) aged ≤6wks. in patients with a genetic diagnosis of SMA & 2 or 3 copies of SMN2
• The trial met its 1EP & 2EPs i.e 100% of patients achieved event-free survival without respiratory or nutritional support & sit independently for ≥30 seconds- including 79% achieved milestone within WHO window of normal development.- 64% walk independently- 100% achieved or maintained CHOP INTEND score of ≥58
• The 82% children in the P-III STR1VE-EU trial for Zolgensma showed improvements in motor function- not observed in the natural history of SMA Type 1

 Ref: Globe Newswire | Image: Novartis