WHY SPINRAZA/PRESYMPTOMATIC STUDY

Supportive study: NURTURE

Who: 25 infants 6 weeks of age and younger who had not yet shown symptoms of SMA


Study time: This supportive study is ongoing


Primary outcomes: Time to death or respiratory intervention


Secondary outcomes: The effect SPINRAZA has on reaching WHO motor milestones


Limitations:Small number of participants. The study is open-label.


Safety: Consistent with the SPINRAZA prescribing information

100% (25/25) of presymptomatic infants were alive without permanent
ventilation after a median follow-up of 2.9 years in the study.

The majority of presymptomatic infants achieved the following WHO motor milestones:

100%

25 out of 25 were
sitting without support

92%

23 out of 25 were
walking with assistance

88%

22 out of 25 were
walking independently

The natural history of SMA
shows that infants with
Type 1 SMA would not
survive
or would require
permanent ventilation by
13.5 months of age.

a child with presymptomatic SMA who is being treated with SPINRAZA

“It's just great seeing him do the things that he's able to do.”

—Christine, Camden's Mom

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