Shots:  

• The P-III STR1VE-EU study involves assessing Zolgensma (IV) in patients with SMA Type 1 aged <6mos. at the time of gene therapy- with 1 or 2 copies of the SMN2 backup gene and have bi-allelic SMN1 gene deletion or point mutations
• Results: 65.6% achieved motor milestones not observed in the natural history of SMA Type 1- 66.7% were free of feeding support- 93.9% are able to swallow thin liquids- 30.3% required feeding support- 27.3% required ventilatory support; 97% patients in ITT population survived event-free- including 93.8% & 56.3% who could have reached 10.5mos. & 13.6 mos. of age respectively
• New interim P-III STR1VE-EU data presented at WMS support the clinical evidence demonstrating a consistent- transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA

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