*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
age 4½ years*Infantile-onset (Type I) SMA
treated with SPINRAZA®
~5 years as of 12/18
1. SPINRAZA® Summary of Product Characteristics.