The gene therapy Zolgensma (onasemnogene abeparvovec) did not cause any serious long-term safety problems when used in children with type 1 spinal muscular atrophy (SMA) in a real-world study from the U.K., and most treated patients experienced improvements in motor function. “This is one of the largest studies…
Blood levels of troponin-I, a marker for heart muscle damage, are best measured before and after gene therapy in each newborn with spinal muscular atrophy (SMA), a new study concluded. Although elevated troponin-I has been associated with SMA gene therapy, levels above the reference values generated from adults were…
Modified surgery to correct a severe abnormal curvature of the spine in people with spinal muscular atrophy (SMA) allowed for reliable access to the spinal canal for Spinraza (nusinersen) injections, a small study shows. Spinraza, guided by ultrasound, was successfully administered through a small hole created in the…
A type of surgery to correct scoliosis, a spine deformity, was found to be generally safe and effective in young people with spinal muscular atrophy (SMA) type 1, a small study shows. Called growth friendly instrumentation, the procedure had been well established for people with SMA type…
Treatment with Spinraza (nusinersen) led to modest improvements in motor function for most children with type 1 spinal muscular atrophy (SMA) who were on permanent ventilation in a Korean study. The results are particularly noteworthy because Spinraza is approved in South Korea only for those patients who are…
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…
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…
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