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
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.