adults with SMA

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…

SMA-specific tests needed to better measure fatigue in adults: Survey

Existing tools for assessing fatigue may not be enough to capture differences in fatigue severity between adults with different types of spinal muscular atrophy (SMA), according to a recent survey conducted by patient advocacy group Cure SMA. SMA patients generally had more fatigue than adults in the general…