Heart marker troponin-I best tested in each SMA newborn: Study
Blood levels of troponin-I, a marker for heart muscle damage, are best measured before and after gene therapy in each…
Articles by Steve Bryson, PhD
Modified scoliosis surgery helps Spinraza treatment in severe cases
Modified surgery to correct a severe abnormal curvature of the spine in people with spinal muscular atrophy (SMA) allowed…
Consistent DMT access is major concern for caregivers in Canada
Consistent access to disease-modifying therapies (DMTs) is a major concern for parent caregivers of children with spinal muscular…
AI-based tool to assess muscles, nerves shows promise as biomarker
A newer assessment method known as dEMB, for decomposition electromyography — which uses artificial intelligence (AI) tools to measure nerve…
Early DMTs may slow scoliosis progression in SMA type 2
Disease-modifying therapies (DMTs) may slow the progression of an abnormal curvature of the spine in people with spinal muscular…
Newborns treated with Evrysdi sit unassisted after 1 year
After a year of treatment with oral Evrysdi (risdiplam), 80% of babies with pre-symptomatic spinal muscular atrophy (SMA)…
Antibody levels affecting Zolgensma transient in SMA children
Elevated levels of neutralizing antibodies against AAV9, which can reduce the efficacy of the gene therapy Zolgensma (onasemnogene abeparvovec),…
Smartphone tool able to capture daily SMA motor function changes
A novel smartphone sensor-based assessment tool is able to capture day-to-day changes in motor function in people with spinal…
Gains in health-related quality of life seen with Spinraza in study
Treatment with Spinraza (nusinersen) was found to improve psychosocial function — the day-to-day ability to contend with environmental and…
Telemedicine use high in SMA community, Cure SMA survey finds
About 80% of the people living with spinal muscular atrophy (SMA) in the U.S. have used telemedicine at least…