ENDEAR: Study design

Study design and patient characteristics

  • A Phase III, randomised, double-blind, sham-procedure controlled study1
  • 121 symptomatic infants ≤7 months of age, diagnosed with SMA (symptom onset before 6 months of age)1
  • Patients were deemed most likely to develop Type I SMA1
  • Patients were randomised 2:1 to either SPINRAZA® or sham-control, with a length of treatment ranging from 6 to 442 days1
  • Median age of onset of clinical signs and symptoms of SMA was 6.5 weeks (SPINRAZA®) and 8 weeks (sham-control)1
  • Median age at first dose: 164.5 days (SPINRAZA®) and 205 days (sham-control)1
Scheme
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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.