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Novartis Presents New Data & Post-hoc Analyses of Zolgensma in Multiple Trials for the Treatment of Spinal Muscular Atrophy at MDA 2022

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Novartis Presents New Data & Post-hoc Analyses of Zolgensma in Multiple Trials for the Treatment of Spinal Muscular Atrophy at MDA 2022

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  • 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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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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