Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
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.
The majority of these presymptomatic infants achieved the following WHO motor milestones:
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.
“It's just great seeing him do the things that he's able to do.”