Daily treatment with Evrysdi (risdiplam) may help stabilize or improve motor function and improve well-being in adults with severe forms of spinal muscular atrophy (SMA), according to a real-world study from the Netherlands. The study showed…
Muscle ultrasound helps assess muscle health and its association with motor function in children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), according to a recent study. While the findings varied by muscle group…
Weak bones in people with spinal muscular atrophy (SMA) may be driven by disrupted muscle-to-bone communication, a study in China showed. The researchers suggested that low levels of SMN protein — a hallmark of SMA…
Treatment with Evrysdi (risdiplam)Â generally stabilized motor and respiratory function for up to 2.5 years in adults with spinal muscular atrophy (SMA), according to a real-world study in Slovenia. The study included 11 adults with SMA…
Blood levels of tiny cell-released particles called extracellular vesicles (EVs) could serve as a tool to track disease progression and treatment response in people with spinal muscular atrophy (SMA), a small study suggests. Researchers discovered that these…
People with spinal muscular atrophy (SMA) show high long-term adherence to treatment with Evrysdi (risdiplam), with several patients remaining on the oral therapy after three years, according to a real-world study in Spain. The observational study,…
Children with spinal muscular atrophy (SMA) who have two or fewer copies of the SMN2 gene — a feature typically associated with more severe disease — are more likely to start treatment early and reach key…
Spinal muscular atrophy (SMA) is more common in wealthier countries across the globe, likely reflecting increased access to diagnostic tools, newborn screening, and modern treatments. That higher prevalence — the total proportion of a population…
The antipsychotic medication haloperidol may help treat spinal muscular atrophy (SMA) by increasing SMN protein levels and enhancing protective effects in nerve cells, a study found. Haloperidol boosted nerve cell survival, reduced neuroinflammation, and improved motor function…
Treatment with Evrysdi (risdiplam) improved motor function and quality of life in children and adults with spinal muscular atrophy (SMA), a real-world study in Hong Kong shows. The findings, from 34 patients followed for up…
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