Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial
Treatment duration: 13 months
Participants: 121 patients with early-onset SMA aged ≤7 months at time of first dose
Primary endpoints: Survival without the need for permanent ventilation and proportion of patients meeting the criteria for motor milestone responder using HINE-2
Safety: The most common side effects were lower respiratory infection (55%) and constipation (35%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)
Learn more about the mobility measures used in the SPINRAZA clinical trials.
A treatment responder was defined as any patient achieving an improvement in more categories of motor milestones than worsening according to HINE-2.1
HINE-2=Hammersmith Infant Neurological Examination Section 2, motor milestone portion.
The ENDEAR Trial concluded early because of the results of the interim analysis, hence why not all patients were assessed at Day 394.
SEM=standard error of the mean.
“Whether she can orcan’t do something, she is going to try.”