More work is needed to expand access to the gene therapy Zolgensma (onasemnogene abeparvovec) for people with spinal muscular atrophy (SMA) living outside of wealthy countries like the U.S. or Australia. That’s the argument made by researchers in the paper, “Gene therapy-based strategies for spinal muscular…

Michael, my patient who has spinal muscular atrophy (SMA), has recently transferred into a new wheelchair and was experiencing significant discomfort in his right hip while seated. We performed several different modalities and activities on Michael’s tailbone to increase his comfort. A combination of these adjustments was…

People with spinal muscular atrophy (SMA) cleared acetaminophen, a common ingredient in pain medications, slower than healthy people, but this was generally not associated with evidence of liver toxicity for most patients, a study shows. Only one of 12 SMA patients developed elevations in markers of liver dysfunction, but…

Disruptions to norepinephrine (NE), a brain signaling molecule, appear to be due to SMN protein loss and could contribute to disease progression in spinal muscular atrophy (SMA) patients, according to recent research. Levels of the molecule, as well as the enzymes needed to produce it, were deficient in a…

Adults with spinal muscular atrophy (SMA) type 3 showed deficits in certain areas of cognition, particularly executive function or the ability to regulate one’s own actions, according to a recent study in Chinese patients. Data from the small analysis suggest that these issues tend to be more pronounced in…

Consistent access to disease-modifying therapies (DMTs) is a major concern for parent caregivers of children with spinal muscular atrophy (SMA) in Canada, a study reported. Barriers to such treatments, as described by caregivers, included variable regulatory approvals across different Canadian provinces, high costs, and insufficient healthcare infrastructure. “Our…