New results from the NURTURE study show ongoing benefits for people with spinal muscular atrophy (SMA), when treated with Spinraza (nusinersen).
NURTURE, the longest trial of pre-symptomatic people with SMA Type 1, shows progressive gains in motor function compared to the natural course of the disease.
As of February 2020, all children treated were alive and remained free of permanent ventilation. In the absence of treatment, the majority would not live beyond two years. The median age of children in the trial was 3.8 years.
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