New Zolgensma data demonstrate age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline

  • All children (100%) treated presymptomatically in the SPR1NT two-copy cohort survived without respiratory or nutritional support, and sat independently for ≥30 seconds, most (11/14) within the WHO window of expected normal development
  • The majority of children (82%) treated in STR1VE-EU achieved developmental motor milestones not observed in the natural history of SMA Type 1, including patients with more severe disease
  • More than 1,200 patients have now been treated with Zolgensma globally across …
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