EMBRACE: Study design

Study design and patient characteristics

  • A Phase II, randomised, double-blind, sham-controlled study1
  • Patients were symptomatic and diagnosed with infantile-onset (n=13) or later-onset SMA (n=8)1
  • Patients were ineligible for participation in ENDEAR or CHERISH due to screening age or SMN2 copy number1
  • Patients had 2 or 3 SMN2 gene copies1
Scheme
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Cameron
age 4½ years*
Infantile-onset (Type I) SMA
treated with SPINRAZA®
~5 years as of 12/18

*Age at time of photo shoot.

References:
1. SPINRAZA® Summary of Product Characteristics.