ENDEAR: Motor milestone results

SPINRAZA® improves achievement of motor milestones in infantile-onset (Type I) SMA1

  • Primary endpoint: At the final analysis, 51% (n=37) of SPINRAZA®-treated patients had met the pre-specified motor milestone responder criteria compared with 0% (n=0) of sham-control patients (p<0.0001)*1

Proportion of patients with improvement or worsening in total HINE-2 motor milestone score1

*Assessed at the later of Day 183, Day 302, and Day 394 Study Visit.1 A motor milestone responder was defined according to the Hammersmith Infant Neurological Examination (HINE) Section 2: ≥2 point increase (or maximal score) in ability to kick, or ≥1 point increase in the motor milestones of head control, rolling, sitting, crawling, standing or walking, and improvement in more categories of motor milestones than worsening.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.