Children diagnosed with spinal muscular atrophy (SMA) and started on Spinraza (nusinersen) as presymptomatic newborns safely continue to…
Articles by Lindsey Shapiro, PhD
A histone deacetylase (HDAC) enzyme inhibitor called AR42 was found to prolong survival and ease motor symptoms in a mouse…
A growing number of young children with spinal muscular atrophy (SMA) type 1 who started taking Evrysdi (risdiplam)…
Add-on treatment with Scholar Rock’s muscle-targeted therapy apitegromab continues to stabilize or improve motor function for young spinal…
Researchers have developed new patient-derived cell models of spinal muscular atrophy (SMA) caused by SMN1 mutations as well as the more…
Muscle abnormalities observed with ultrasound imaging correlated with motor function in people with spinal muscular atrophy (SMA) in a…
More than a year of treatment with Spinraza (nusinersen) did not result in clinically meaningful changes in cognition for…
Researchers have developed a new mouse model that exhibits the features of spinal muscular atrophy (SMA) with progressive myoclonic…
The adoption of a newborn screening (NBS) program for spinal muscular atrophy (SMA) in England would reduce healthcare costs…
The overactivity of a specific signaling pathway may contribute to the abnormalities in clearing cellular waste that are commonly seen…