Considering SPINRAZA? Discover the data and talk to your doctor to learn more.

Improvements are possible with SPINRAZA

When started earlier, SPINRAZA may help children progress on WHO motor milestones consistent with normal development.

Supportive study: NURTURE

Who: 25 infants aged 6 weeks 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. After a follow-up of over 14 months to 5.7 years, 25/25 infants experienced any AE (mild, moderate, or severe), and no new safety concerns were identified

After at least 14 months of treatment:

At the initial interim analysis performed after all infants had received SPINRAZA for at least 14 months (median, 25 months; range, 14 to 34 months), 100% (25/25) of infants were alive without the need for permanent ventilation, 100% (25/25) were sitting without support, 88% (22/25) were walking with assistance, and 77% (17/22) of infants were walking independently.

After nearly 5 years of follow-up*:

100% (25/25) of presymptomatic infants were alive without permanent ventilation after a median follow-up of 4.9 years in the study. 84% (21/25) never required respiratory intervention

Respiratory intervention is defined as ventilation for ≥6 hours/day continuously for ≥7 days, or tracheostomy.

Permanent ventilation is defined as ventilation for ≥16 hours/day for >21 days in absence of an acute reversible event, or tracheostomy.

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


25 out of 25 were
sitting without support


24 out of 25 were walking
with assistance


23 out of 25 were walking

*At least half of patients were followed for at least 4.9 years.

Additional Data

After ~3 years of follow-up§

88%22 out of 25
able to suck/ swallow well

based on maximal HINE-1 score at the
day 778 visit

After ~4 years of follow-up

84%21 out of 25
never required tube feeds

92%23 out of 25 never needed to
suction excess saliva or drool

based on caregiver assessment of swallowing difficulty during mealtime over a period of 7 days

§At least half of patients were followed for at least 3.8 years.

A child with
presymptomatic SMA who
is being treated with

“For Camden to do what he is able to
do, we are just so thankful.” – Michael, Camden’s dad

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