*NURTURE study interim analysis data cutoff date: May 15 2018; ENDEAR-SHINE integrated analysis data cutoff date: June 30 2017. CS3A end-of-study data for the cohort of infants with 2 SMN2 copies. ENDEAR participants with 2 SMN2 copies in the intention-to-treat population. ENDEAR data were windowed into intervals based on time from baseline. For each study, n≥5 are plotted.1
Stock image (not a real patient).
References:
1. SPINRAZA® Summary of Product Characteristics.