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…
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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…
Existing tools for assessing fatigue may not be enough to capture differences in fatigue severity between adults with different types of spinal muscular atrophy (SMA), according to a recent survey conducted by patient advocacy group Cure SMA. SMA patients generally had more fatigue than adults in the general…
Children with spinal muscular atrophy (SMA) type 1 may be at an increased risk of developing kidney stones, suggesting the condition may be more common than previously thought, according to a small study in Turkey. It’s possible that nephrolithiasis — the medical term for kidney stones — had been…
A newer assessment method known as dEMB, for decomposition electromyography — which uses artificial intelligence (AI) tools to measure nerve and muscle function — strongly correlated with measures of physical function and was able to distinguish people with spinal muscular atrophy (SMA) who were able to walk from those…
Children with spinal muscular atrophy (SMA) generally have poor bone health, including low bone mineral density and reduced bone formation and remodeling, a study indicates. The effect on bone health is more pronounced in children with worse motor function, but as treatment with Spinraza (nusinersen) improved motor function…
Certain RNA molecules may be used as biomarkers to assess response to treatment with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA), according to a recent study. These RNA (which stands for ribonucleic acid) molecules are found in the cerebrospinal fluid (CSF), the liquid surrounding the brain…
How spinal muscular atrophy (SMA) manifests among people who carry four copies of the SMN2 gene varies, ranging from type 2 to type 4, with functional abilities showing a tendency to decline with age, a study in Italy finds. Understanding how the disease progresses over time and what influences…
About a third of adults with spinal muscular atrophy (SMA) show signs of poor, and abnormal, lung function, according to new analyses that found breathing problems were more likely in patients with severe disease — including those with type 2 SMA and those without an ability to walk. Lower…
