Study design and patient characteristics
- A Phase III, randomised, double-blind, sham-procedure controlled study1
- 126 symptomatic patients with later-onset SMA (symptom onset after 6 months of age)1
- Patients in this study were deemed most likely to develop Type II or III SMA1
- Patients were randomised 2:1 to either SPINRAZA® or sham-control, with a length of treatment ranging from 324 to 482 days1
- Median age of onset of clinical signs and symptoms of SMA: 11 months1
- Median age at screening: 3 years1
- Most patients (88%) have 3 copies of the SMN2 gene (8% have 2 copies, 2% have 4 copies, and 2% have an unknown copy number)1
Ruby
age 6 years*Later-onset (Type II) SMA
treated with SPINRAZA®
~4 years as of 9/18
*Age at time of photo shoot.
References:
1. SPINRAZA® Summary of Product Characteristics.