Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing

  • Interim SPR1NT data showed presymptomatic babies with SMA treated with Zolgensma® (onasemnogene abeparvovec-xioi) soon after birth achieved age-appropriate motor milestones
     
  • In addition to meeting both co-primary efficacy endpoints, nine of 22 patients in the completed pivotal STR1VE-US study demonstrated the “ability to thrive,” a stringent composite endpoint remarkable compared to untreated children with SMA Type 1
     
  • Ongoing START long-term follow-up study (cohort 2) demonstrated sustained durability of Zolgensma, …
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AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2

  • SMA Type 2 patients between two and five years of age who received Dose B met the primary efficacy endpoint with a remarkable mean increase of 6.0 points in HFMSE scores from baseline to month 12, twice the clinically meaningful threshold, as measured by a validated, widely utilized and highly accepted scale developed specifically for older patients with SMA
     
  • Nearly all patients (92%) in this cohort achieved a clinically meaningful ≥3-point increase during the study period, demonstrating a consistent response following gene therapy
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