WHY SPINRAZA/EARLY-ONSET STUDY
Pivotal study: ENDEAR
Who: 121 children 7 months of age and younger with Type 1 (early-onset) SMA
Study time: 13 months
Primary outcomes:
- Time to death or use of permanent assisted ventilation
- The proportion of responders (or number of children) who showed an improvement in motor milestones according to HINE-2
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%).
Those with Type 1 SMA showed improvements in motor milestones that are rarely, if ever, achieved in untreated children
51% vs 0% untreated were motor milestone responders according to HINE-2 at 13 months. Motor milestones included:
“Whether she can or can't do something, she's going to try.”