Systemic Treatment May Be More Effective for SMA, Mouse Study Suggests
Restoring levels of the SMN protein exclusively in nerve cells through gene therapy only partly eased symptoms and lengthened…
Articles by Marta Figueiredo, PhD
Risdiplam Continues to Show Promise for Treating SMA, Data Show
Risdiplam continues to sustain high levels of the SMN protein and improve motor function in children and young adults with…
96% of Children Given Spinraza as Newborns Able to Walk in NURTURE Trial
Spinraza (nusinersen) continues to safely and effectively stop disease progression in children with spinal muscular atrophy (SMA) who…
SMA Less Common Than Expected Among Newborns Screened in NY
The frequency with which spinal muscular atrophy (SMA) is found in newborns may be lower than previously thought, according to…
Heart Problems in SMA May Be Tied to Calcium Dysregulation, Study Suggests
Heart problems associated with spinal muscular atrophy (SMA) may be caused partially by calcium dysregulation in heart muscle cells in…
Spinraza Meets Most Therapeutic Expectations of Adult SMA Patients, Real-World Study Shows
Spinraza (nusinersen) improves muscle strength, endurance, and independence in adults with spinal muscular atrophy (SMA), meeting most of their…
Lung Ultrasound Reliable Option to Chest X-rays for Monitoring Children
Lung ultrasound is a safe and reliable bedside tool to detect atelectasis (partial lung collapse) early in children with neuromuscular…
Risdiplam Defies SMA ‘Natural History’ in Type 1 Infants, FIREFISH Data Show
Roche’s experimental oral treatment risdiplam can safely and effectively halt disease progression in infants with type 1…
SBMA Severity Associated With Altered Muscle, Not Neuronal, Biomarkers, Study Shows
Blood levels of biomarkers of muscle mass and damage — rather than nerve cell damage — are significantly altered in…
Risdiplam’s Potential Likely Due to Systemic Nature and Specificity, Roche Says in Interview
The systemic nature and high specificity of risdiplam — an oral treatment under review in the U.S. to…