Spinraza®: positive long-term data reported in pre-symptomatic infants

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...