CS2 & CS12: Study design

Study design and patient characteristics

  • CS2 and CS12 are open-label studies of patients with later-onset SMA1
  • This analysis included 28 patients who received their first dose in Study CS2 and then transferred to the extension phase, Study CS121
  • The studies enrolled patients who were between 2 to 15 years of age at first dose1
  • Of the 28 patients, 3 were at least 18 years of age at their last study visit1
  • 1 out of 28 patients had 2 SMN2 gene copies, 21 had 3 copies, and 6 had 4 copies1

age 20 years*
Later-onset (Type III) SMA
treated with SPINRAZA®

*Age at time of photo shoot.

1. SPINRAZA® Summary of Product Characteristics.