*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.1
†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
Stock image (not a real patient).
1. SPINRAZA® Summary of Product Characteristics.