First-line gene therapy may offer better sustained results in SMA
First-line Zolgensma (onasemnogene abeparvovec-xioi) outperformed first-line Spinraza (nusinersen) in achieving a more sustained clinical response and reducing the…
Articles by Steve Bryson, PhD
SMA-1 kids can survive lung crisis with standard protocols: Study
Children with spinal muscular atrophy (SMA) type 1 can survive an episode of pediatric acute respiratory distress syndrome (pARDS),…
Coordination lacking in transition from SMA pediatric to adult care
In a new interview-based study, adults with spinal muscular atrophy (SMA) reported a lack of care coordination during their…
SMA gene therapy GC101 safe, effective in children, adolescents
Gene therapy GC101 safely improves or stabilizes motor function in children and adolescents with spinal muscular atrophy (SMA) types…
Nerve proteins may serve as biomarkers for SBMA progression
Adults with spinal and bulbar muscular atrophy (SBMA) have elevated levels of neurofilament light (NfL) chain and glial fibrillary…
New ASAH1 mutation found to cause rare SMA type in boy, 4
A novel mutation in the ASAH1 gene was identified as the cause of spinal muscular atrophy with progressive myoclonic…
Digital PCR for SMA diagnosis faster, less costly than set methods
Digital PCR, which simultaneously detects SMN1 gene defects and the number of SMN2 copies to diagnose spinal muscular atrophy…
DySMA on swallowing in healthy children offers SMA comparison
Not all healthy children acquire full feeding skills by 2 years, according to a study that applied an instrument called…
In SMA, Spinraza boosts signals in nerves that supply muscles
Motor improvements in adults with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) paralleled a boost in the…
Pregnancies reported for partners of 3 late-onset SMA men on Evrysdi
Three men with late-onset spinal muscular atrophy (SMA) undergoing treatment with the approved therapy Evrysdi (risdiplam) were able…