Altered body fat, metabolic disturbances common in SMA adults
Adults with spinal muscular atrophy (SMA) showed alterations in body composition and metabolic disturbances, including changes in blood sugar…
Articles by Lindsey Shapiro, PhD
Newborns given Evrysdi reaching key milestones after 2 years
Newborns diagnosed with spinal muscular atrophy (SMA) who were treated with Evrysdi (risdiplam) before the onset of symptoms…
High dose of SMA treatment Spinraza shows benefits: Trial data
An experimental high-dose regimen of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) is showing motor benefits for previously…
Premature babies, diagnosed via screening, treated for SMA
Babies born prematurely were diagnosed with spinal muscular atrophy (SMA) through newborn screening programs (NBS) in Germany and, in…
Changes in normal brain structure seen on MRI scans of SMA patients
Brain abnormalities visible on MRI scans were seven times more likely in people with spinal muscular atrophy (SMA) than…
More nuanced look at SMA upper limb function offered in analysis
Untreated patients with spinal muscular atrophy (SMA) type 2 showed more changes in their ability to perform upper limb…
Spinraza treatment improves motor function in older SMA patients
Treatment with Spinraza (nusinersen) generally stabilized or improved motor function for older patients with spinal muscular atrophy (SMA),…
High-dose Spinraza treatment shown to safely benefit SMA infants
Treatment with an investigational high-dose regimen of Spinraza (nusinersen) was well tolerated and significantly improved motor function in infants…
Global metabolic disruption found in children with SMA in new study
Children with spinal muscular atrophy (SMA) exhibit widespread metabolic disruption — changes that affect the body’s metabolism — relative…
Double gene therapy leads to lasting benefits in SMA mouse model
A combination genetic therapy approach designed to restore more normal activity of the SMN1 gene improved motor function and prolonged survival…