In a pivotal trial, SPINRAZA achieved significant results in important milestones.
Who: 121 children aged 7 months and younger with Type 1 (early-onset) SMA
Study time: 13 months
Safety: The most common side effects were lower respiratory infection (55%) and constipation (35%). Serious adverse reactions of collapsed lung (atelectasis) were more frequent in the SPINRAZA-treated group (18%) than in the control group (10%).
“Whether she can or can't do
something, she's going to try.”