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