Scholar Rock plans to resubmit its application to the U.S. Food and Drug Administration (FDA) seeking approval for…
Articles by Steve Bryson, PhD
Damage to blood vessels in the spinal cord may play a role in nerve cell loss in spinal muscular…
Children with spinal muscular atrophy (SMA) who are nonambulatory, or unable to walk, have weaker upper leg bones —…
First-line Zolgensma (onasemnogene abeparvovec-xioi) outperformed first-line Spinraza (nusinersen) in achieving a more sustained clinical response and reducing the…
Children with spinal muscular atrophy (SMA) type 1 can survive an episode of pediatric acute respiratory distress syndrome (pARDS),…
In a new interview-based study, adults with spinal muscular atrophy (SMA) reported a lack of care coordination during their…
Gene therapy GC101 safely improves or stabilizes motor function in children and adolescents with spinal muscular atrophy (SMA) types…
Adults with spinal and bulbar muscular atrophy (SBMA) have elevated levels of neurofilament light (NfL) chain and glial fibrillary…
A novel mutation in the ASAH1 gene was identified as the cause of spinal muscular atrophy with progressive myoclonic…
Digital PCR, which simultaneously detects SMN1 gene defects and the number of SMN2 copies to diagnose spinal muscular atrophy…