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:

63% reduced risk of mortality in the SPINRAZA group

a child with early-onset SMA who is being treated with SPINRAZA

“Whether she can or can't do something, she's going to try.”

—Sofia’s Mom

Your healthcare provider will monitor for signs of bleeding
complications or kidney damage while you are taking SPINRAZA.