Being on Spinraza (nusinersen) for up to three years results in a gain in motor function for children with spinal muscular atrophy (SMA) type 2, with the youngest children benefiting the most from treatment, a small study has found. “It is therefore important to start the treatment as…
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Revvity has launched its EONIS Q system for faster and simplified newborn molecular screening of spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID), the company announced. According to Revvity, this easy-to-use platform reduces the complexity and costs for laboratories, so they can expand newborn screening capabilities.
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…
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…
NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA), was seen to be generally safe and well tolerated in healthy adults who participated in an initial clinical trial. Results of the Phase 1 study (ACTRN12622000964718) were presented during the 2023 International Kennedy’s…
A drop in the levels of four proteins in the cerebrospinal fluid (CSF) may be a marker of response to treatment with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), a real-world data study suggests. The predictive value of the four-protein panel — which includes neurofilament light chain,…
Treatment with Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec) led to increases in the electrical activity of motor neurons — the specialized nerve cells that control voluntary movement — for two young children with type 1 spinal muscular atrophy (SMA), according to a new report. Findings from tests…
