Spinraza has been widely used since its U.S. approval in 2016 and is cleared to treat both pediatric and adult patients with SMA.
Yet Zolgensma, with its promise of long-lasting benefit, has given families of the 450 to 500 newborns and young infants diagnosed with SMA in the U.S. each year another choice. About 100 patients each quarter are now being treated with Zolgensma, which is designed to correct the genetic defect that leads to the disease.
