Children with spinal muscular atrophy (SMA) can still experience swallowing difficulties despite early treatment with disease-modifying therapies, a small…
Articles by Steve Bryson, PhD
In a small real-world study that tracked adults with spinal muscular atrophy (SMA) treated with Evrysdi (risdiplam) for…
A previously unknown breathing problem — called pseudo-obstructive sleep-disordered breathing — has been discovered among people with spinal muscular…
A team of scientists in Iowa have created a super minigene — a small gene fragment used as a tool…
Despite concerns about long-term efficacy and safety, gene therapy was the first choice treatment for most parents of children with…
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…