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Treatment with Spinraza (nusinersen) improved motor function in children, adolescents, and adults with spinal muscle atrophy (SMA) who were unable to walk without assistance, according to a four-year real-world analysis. Moreover, nine of the 12 patients who underwent continuous evaluations while on Spinraza over the four years…
Researchers have designed a way to help therapies like Spinraza (nusinersen) — approved for spinal muscular atrophy (SMA) — better access the brain and spinal cord when delivered into the bloodstream. In a recent report, the scientists linked a molecule similar to Spinraza with small protein fragments…
Most individuals with spinal muscular atrophy (SMA) type 2 experience improvements in physical abilities within a year after starting treatment with Spinraza (nusinersen). However, about 1 in 3 patients had some loss of function in the first year after beginning the approved SMA therapy. That’s according to…
Researchers have developed a new laboratory technique for measuring levels of Spinraza (nusinersen), an approved treatment for spinal muscular atrophy (SMA), in biological fluids. The test is expected to help overcome some limitations of other approaches now in use, and may therefore aid scientists and physicians when studying the…
In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), subsequent treatment with Spinraza (nusinersen) can improve motor function and may reduce nerve damage. That’s according to interim data from the Phase 4 RESPOND study…
Long-term treatment with Spinraza (nusinersen) was associated with improved or stable motor function in patients across the spinal muscular atrophy (SMA) disease spectrum, according to an analysis of real-world registry data. “To date, this is the largest prospective study over the longest observational period of [Spinraza] therapy…
Difficulties with adequate nutrition and feeding persist among children and adolescents with spinal muscular atrophy (SMA) types 2 and 3 being treated with Spinraza (nusinersen), a Norwegian study reports. Important nutrients, such as protein, fiber, vitamins, and minerals, often were consumed in lower-than-recommended amounts by these patients,…
Throughout 2023, SMA News Today brought you the most up-to-date news on scientific breakthroughs and treatment advances related to spinal muscular atrophy (SMA). These are the top 10 most-read articles we published throughout that year, with a brief reminder of what made them relevant to the SMA community. We…
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…
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,…
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