ENDEAR: Motor function results

SPINRAZA® helps patients with infantile-onset (Type I) SMA improve motor function1

  • 71% (n=52) of SPINRAZA®-treated patients achieved a meaningful ≥4-point improvement in CHOP INTEND scores compared with 3% (n=1) of sham-control patients (p<0.0001)1

Change in CHOP INTEND score in 78 patients*1

 

*Infants with opportunity for at least a 6-month (Day 183) assessment were included in the analysis; last available assessment of 6-month (Day 183), 10-month (Day 302), or 13-month (Day 394) was used. Shortest bars indicate zero value. Of the 110 infants in the efficacy set, 29 died (SPINRAZA®, n=13; sham-control, n=16) and three withdrew for a reason other than death (SPINRAZA®, n=2; sham-control, n=1) and were not included in this analysis.1

Sofia
age 4½ years*
Infantile-onset (Type I) SMA
treated with SPINRAZA®
~4 years as of 10/18

*Age at time of photo shoot.

References:
1. SPINRAZA® Summary of Product Characteristics.