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%)
*Permanent assisted ventilation was defined as tracheostomy or ventilatory support for ≥16 hours per day for >21 continuous days in the absence of an acute reversible event.
Learn more about the mobility measures used in the SPINRAZA clinical trials.
*Permanent assisted ventilation was defined as tracheostomy or ventilatory support for ≥16 hours per day for >21 continuous days in the absence of an acute reversible event.
†P<0.0001
Responders=patients achieving an improvement in more categories of motor milestones than worsening according to HINE-2.5
HINE-2=Hammersmith Infant Neurological Examination Section 2, motor milestone portion.
SEM=standard error of the mean.
“Whether she can or
can’t do something, she
is going to try.”