The late 2016 introduction of nusinersen (brand name: Spinraza) as a drug therapy for spinal muscular atrophy (SMA) in the United States poses the question: Can SMA be rendered a chronic rather than a progressive condition?

Missy Ball, MT, PT, ATP, said clinical studies of nusinersen used by children and adults have been promising.

“The findings show support for treatment for all types of SMA (especially types 1, 2 and 3) and support early initiation of treatment,” she said. “Results from different studies show improvement in motor function, including ability to sit unassisted, stand or walk when [study participants] would otherwise be unexpected to do so. Also, longevity improved in a greater percentage of patients on Spinraza.”

A November 2017 study published in the New England Journal of Medicine said 51 percent of infants with SMA who were given nusinersen achieved motor-milestone responses, compared to 0 percent of the infants in the control group who didn’t receive nusinersen. The survival rate was also higher in the group that received nusinersen. Researchers suggested that early treatment might be required to fully benefit from the drug’s abilities.