Novartis Presents New Data & Post-hoc Analyses of Zolgensma in Multiple Trials for the Treatment of Spinal Muscular Atrophy at MDA 2022
Shots:
- The 3-copy cohort of P-III (SPR1NT) trial evaluates Zolgensma (IV) in patients aged ≤6 wks. with SMA & 2 or 3 copies of SMN2. The results showed that patients achieved age-appropriate milestones, 93% & 100% walk & stand-alone with 73% & 93% in WHO window of normal development, 100% were free of nutritional & respiratory support with no serious AEs
- A posthoc analysis from the P-I (START) & 2 P-III (STR1VE-EU) & (STR1VE-US) studies of Zolgensma in children with SMA Type 1 achieved or maintained bulbar function, 80% achieved composite EPs of speak, swallow & maintain airway protection
- Additionally, 95% met communication EPs, 92% had 1 occurrence of a normal swallow test, 92% did not report any event
Ref: Novartis | Image: Novartis
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