Study design and patient characteristics
- An open-label study in pre-symptomatic infants genetically diagnosed with SMA, who were enrolled at 6 weeks of age or younger1
- Patients in this study were deemed most likely to develop Type I or II SMA1
- 2 SMN2 gene copies: n=15; 3 SMN2 gene copies: n=101
- Median age at first dose: 22 days1
- An interim analysis was conducted when patients had been on the study for a median of 27.1 months (15.1–35.5 months) and were of a median age at last visit of 26.0 months (14.0–34.3 months)1
- The study is ongoing
Stock image (not a real patient).
References:
1. SPINRAZA® Summary of Product Characteristics.