Final analysis was assessed
on day 394 of the trial.1
Some patients with infantile-onset (Type 1) SMA treated with SPINRAZA achieved new improvements in motor milestones.1,2
*Ability to kick includes achievement of touching toes (n=5).3
Patients with infantile-onset (Type 1) SMA treated with SPINRAZA showed continued improvement in mean total motor milestone score.4
HINE=Hammersmith Infant Neurological
Examination; SEM=standard error of the mean;
SMA=spinal muscular atrophy.
†Excludes voluntary grasp.4
Milestones such as head control and rolling are rarely, if ever, achieved in untreated patients in the natural history of Type 1 SMA.5
Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
3. Data on file. Biogen, Cambridge, MA.
4. Finkel RS, Mercuri E, Darras BT, et al; for the ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy (supplementary appendix). N Engl J Med. 2017;377(18):1723-1732.