SHINE: Interim results - Infantile-onset SMA

ENDEAR-SHINE interim results: Earlier SPINRAZA® treatment gives greatest benefit1

  • 89 patients with infantile-onset (Type I) SMA from ENDEAR enrolled in SHINE1
  • At the interim analysis, the length of SPINRAZA® treatment ranged from 65 to 592 days (median: 289 days)1
  • Motor function improved in all patients, irrespective of whether SPINRAZA® treatment was initiated in ENDEAR or SHINE1
  • The greatest benefit was seen with earlier initiation of SPINRAZA®1

Change in HINE-2 Motor Milestone Score over time (integrated analysis)*1


*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

*Age at time of photo shoot.

1. SPINRAZA® Summary of Product Characteristics.