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

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.

WHY SPINRAZA/EARLY-ONSET STUDY

The effectiveness of SPINRAZA has been studied in the longest clinical trial program in SMA to date.

Pivotal study:
ENDEAR

ENDEAR was a 13-month study in 121 children 7 months of age and younger with Type 1 (early-onset) SMA. It was a randomized, sham-controlled study, which means children were randomly placed into a treated or untreated group.

The study evaluated the effect SPINRAZA has on survival without permanent ventilation and the proportion of “responders,” or children who showed an improvement in motor milestones, according to an assessment scale called HINE-2.

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 9 months.
Motor milestones included:

graphic featuring major milestones children on treatment with SPINRAZA achieved
graphic featuring major milestones children on treatment with SPINRAZA achieved

HINE-2: Section 2 of the Hammersmith Infant Neurological Examination (HINE) is used to assess improvement in motor function. The scale includes activities such as head control, independent sitting, and standing.

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

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.

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