Authors: Sharon Salt, Editor
Biogen (Maidenhead, UK) have announced new results from the NURTURE study, which demonstrated that pre-symptomatic infants treated with Spinraza® (nusinersen) achieved motor milestones not before seen in the natural history of the disease, including 100% of children sitting without support and 88% walking independently.
NURTURE is an ongoing, Phase II, open-label study of 25 pre-symptomatic patients with spinal muscular atrophy (SMA; most likely to develop SMA Type 1 or Type 2) who received their first dose of nusinersen before 6 weeks old.
These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with nusinersen in comparison to the natural history of the disease. These new data also revealed that patients treated with nusinersen had continuous improvement in sitting and walking independently, with an overwhelming majority of patients achieving motor milestones in a normal timeframe.