Treatment with Zolgensma (onasemnogene abeparvovec-xioi) helped children with spinal muscular atrophy (SMA) gain or maintain motor milestones and motor function, a real-world study in Japan found. The RESTORE patient registry (NCT04174157) is assessing the long-term safety and efficacy of Zolgensma in clinical settings across 99 locations in the…
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The migraine medication flunarizine may help protect nerve cells in spinal muscular atrophy (SMA) by correcting abnormal levels of microRNA, small molecules that help regulate gene activity. Researchers identified widespread microRNA abnormalities in cells and in the spinal cords of SMA mice, particularly at the earliest stages of disease.
Healthcare professionals who specialize in managing spinal muscular atrophy (SMA) are prone to putting off tasks related to patient care, likely because they are stressed and exhausted, a study found. A research team in Spain found a strong relationship between burnout and procrastination, suggesting that professional exhaustion may drive…
Parents of children and young adults with spinal muscular atrophy (SMA) reported significant unmet needs, including respite care, mental health support, and community connection, according to an interview study. Three-quarters of the parents also expressed interest in developing a tailored parenting program to help them better care for their…
Treatment with Evrysdi (risdiplam) 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 types 2, 3, and 4. “These results add to the limited database…
Using wearable sensors during clinical tests may help doctors objectively measure changes in movement ability for people with spinal bulbar muscular atrophy (SBMA), a study reports. “These findings support incorporating wearable sensors into clinic assessments and encourage future work using home-based monitoring in larger SBMA cohorts,” researchers wrote. Despite…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to salanersen, a new, more potent Biogen treatment for spinal muscular atrophy (SMA) that’s now in late-stage testing. The treatment candidate, being evaluated in two ongoing Phase 3 clinical trials, is similar to — but…
A combination of genetic testing approaches enabled researchers in Spain to more accurately characterize complex genetic changes that may help explain differences in disease severity in spinal muscular atrophy (SMA), according to a new study. The findings suggest that “combining multiple genetic diagnostic techniques” enables more accurate genetic characterization…
Problems with swallowing, also called dysphagia, remain a concern for many children with spinal muscular atrophy (SMA) type 1 despite available treatments, a real-world study reports. Researchers examined swallowing in patients with SMA type 1 receiving disease-modifying therapies (DMTs). They found that…
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 microscopic particles, which carry cargo between cells, were elevated in untreated SMA…
