real-world study

Zolgensma alone helps children with SMA type 1: Real-world study

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), given as a sole treatment, improves motor skills and preserves lung health and feeding abilities in infants with spinal muscular atrophy (SMA) type 1, according to a real-world study. Despite such benefits to patients treated at a mean age of 7.5 months,…

Adherence to Evrysdi treatment found high in real-world analysis

Spinal muscular atrophy (SMA) patients in the U.S. exhibited high rates of adherence to their prescribed regimen of Evrysdi (risdiplam) over a year, according to a recent analysis of pharmacy prescription fill data. “High adherence to and persistence with [Evrysdi] were observed among patients with SMA over…

In real world, Evrysdi stabilizes, improves motor skills in SMA adults

In a small real-world study that tracked adults with spinal muscular atrophy (SMA) treated with Evrysdi (risdiplam) for more than 18 months, most patients saw their motor function remain stable or even improve. Nearly all of the study’s participants reported being generally satisfied with the approved SMA medication…

Zolgensma may safely benefit older, heavier children with SMA: Study

The gene therapy Zolgensma (onasemnogene abeparvovec) did not cause any serious long-term safety problems when used in children with type 1 spinal muscular atrophy (SMA) in a real-world study from the U.K., and most treated patients experienced improvements in motor function. “This is one of the largest studies…