Despite concerns about long-term efficacy and safety, gene therapy was the first choice treatment for most parents of children with…
Articles by Steve Bryson, PhD
The start of a newborn screening (NBS) program for spinal muscular atrophy (SMA), allowing early treatment for infants found…
Blood levels of troponin-I, a marker for heart muscle damage, are best measured before and after gene therapy in each…
Modified surgery to correct a severe abnormal curvature of the spine in people with spinal muscular atrophy (SMA) allowed…
Consistent access to disease-modifying therapies (DMTs) is a major concern for parent caregivers of children with spinal muscular…
A newer assessment method known as dEMB, for decomposition electromyography — which uses artificial intelligence (AI) tools to measure nerve…
Disease-modifying therapies (DMTs) may slow the progression of an abnormal curvature of the spine in people with spinal muscular…
After a year of treatment with oral Evrysdi (risdiplam), 80% of babies with pre-symptomatic spinal muscular atrophy (SMA)…
Elevated levels of neutralizing antibodies against AAV9, which can reduce the efficacy of the gene therapy Zolgensma (onasemnogene abeparvovec),…
A novel smartphone sensor-based assessment tool is able to capture day-to-day changes in motor function in people with spinal…