6MWT=6-Minute Walk Test; SE=standard error.
All nonambulatory patients with Type 3 SMA reached the maximum Upper Limb Module score of 18 points by day 350 and maintained this score through day 1150.1
Among the nonambulatory patients who were unable to walk at baseline:
1 of 11 patients with Type 2 SMA gained the ability to walk for the first time
2 of 4 patients with Type 3 SMA regained the ability to walk that they had previously lost
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
1. Darras BT, Chiriboga CA, Iannaccone ST, et al; on behalf of the ISIS-396443-CS2/ISIS-396443-CS12 Study Groups. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies [published online April 24, 2019]. Neurology. doi:10.1212/WNL.0000000000007527.
2. Data on file. Biogen, Cambridge, MA.