Drug can safely and effectively help infants with SMA gain muscle function study shows

More than half of the babies with infantile-onset spinal muscular atrophy (SMA) who were treated with nusinersen (Spinraza) gained motor milestones, compared to none of the babies in the control group. Infants treated with the drug also had 63 percent lower risk of death. These final results from the 13-month, international, randomized, multicenter, sham procedure-controlled, phase 3 trial called ENDEAR were published in the New England Journal of Medicine.