Pediatric:
Initiation Criteria
1. Pre-symptomatic patients with 2 or 3 copies of the SMN2 gene,
OR
2. Had the disease for <6 months, 2 copies of SMN2, and symptom onset after the first week after birth and on, or before seven months of age.
OR
3. Patients under the age of 18 with symptom onset > 6 months of age, and never achieved the ability to walk independently. †
Stopping Criteria
1. For those pre-symptomatic at initiation: no improvement/maintenance on HINE-2, CHOP INTEND, or HFMSE, OR
2. For those symptomatic at initiation: no maintenance in HINE-2, CHOP INTENT, or HFMSE, OR
3. Permanent invasive ventilation required