Study: Phase 3, multicenter, double-blind, randomized (2:1), sham-controlled
Treatment duration: 13 months
Participants: 121 patients with early-onset SMA aged ≤7months 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: Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)
aPermanent 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.
85% SPINRAZA treated vs 61% untreated survived; HR=0.37; P=0.004
Learn more about the mobility measures used in the SPINRAZA clinical trials.
aPermanent 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.
85% SPINRAZA treated vs 61% untreated survived; HR=0.37; P=0.004
bP<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.
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