• At the interim analysis, 100% of patients (25 of 25) were alive and without permanent ventilation*¹
• Of the 16 patients who reached the Day 700 visit at the interim analysis, 64% (7 of 11) patients with 2 SMN2 gene copies and 0% (0 of 5) with 3 SMN2 copies met the protocol-defined criteria of clinically manifested SMA; all 16 patients were gaining weight and achieving WHO milestones inconsistent with Type I SMA^†1

*The primary endpoint, time to death or respiratory intervention (defined as invasive or non-invasive ventilation for ≥6 hours/day continuously for ≥7 consecutive days or tracheostomy), could not be estimated as there were too few events.<sup>1

†</sup>The protocol-defined criteria for clinically manifested SMA included age-adjusted weight below the fifth WHO percentile, a decrease of ≥2 major weight growth curve percentiles, the placement of a percutaneous gastric tube, and/or the inability to achieve expected age-appropriate WHO milestones (sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone and walking alone).¹