Cure SMA: Children on Nusinersen More Likely to Achieve Milestones

A new study published in the New England Journal of Medicine found that infants who have Type 1 spinal muscular atrophy (SMA) and took the drug nusinersen (brand name: Spinraza) were far more likely to hit motor milestones than infants with SMA who were not given the treatment.

Researchers used the Hammersmith Infant Neurological Examination to determine improvements in motor milestone categories, and they also tracked when study participants needed permanent ventilation and/or when they died.

The study showed that 51 percent of infants on Spinraza “were motor milestone responders, compared to untreated infants,” none of whom achieved motor milestones, according to a news announcement from Cure SMA.

There was also a “statistically significant 47 percent reduction in the risk of death or use of permanent assisted ventilation,” the announcement said.

Cure SMA said Spinraza “demonstrated a favorable benefit-risk profile.”

Last December, nusinersen became the first drug therapy FDA approved to treat SMA, which is the world’s leading genetic cause of death in infants.

About the Author

Laurie Watanabe is the editor of Mobility Management. She can be reached at

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