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…
Far-field potentials (FFPs), electrical signals recorded with surface electrodes after nerve stimulation, may help track disease progression in people with spinal muscular atrophy (SMA), a study suggests. The strength of these signals, or FFP amplitude, was significantly…
A home-based respiratory physiotherapy program focused on breathing exercises and airway clearance techniques can significantly improve quality of life for young people with spinal muscular atrophy (SMA) who are receiving treatment with Spinraza…
Children with spinal muscular atrophy (SMA) showed trends suggesting that nutritional status may be linked to motor function, according to a study in Poland that tracked 38 children over one year. Researchers found…
Even when treated early with disease-modifying therapies (DMTs), infants and toddlers with spinal muscular atrophy (SMA) do not develop swallowing skills at the same pace or to the same level as healthy children. That’s according…
Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a…
Treatment with Spinraza (nusinersen) led to sustained improvements in motor function in adults with spinal muscular atrophy (SMA), and showed a manageable safety profile, according to a nationwide study in South Korea. Among 19 adults, researchers…
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