a child with presymptomatic SMA who is being treated with SPINRAZA
a child with presymptomatic 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/PRESYMPTOMATIC STUDY

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

Supportive study:
NURTURE

NURTURE is an ongoing study in 25 infants 6 weeks of age and younger who have not yet shown symptoms of SMA. The study is evaluating the effect SPINRAZA has on survival without permanent ventilation and on motor function development.

Limitations include: These results are from an ongoing study, which had a small participant size, and the study is open-label, which means all infants in this study receive SPINRAZA.

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 these 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

WHO=World Health Organization.
WHO motor milestones: The World Health Organization motor milestones are a set of six milestones, such as sitting without support, hands-and-knees crawling, and walking alone, expected to be achieved by 2 years of age in healthy children.

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.