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A two-week course of transcutaneous spinal cord stimulation (tSCS), a noninvasive method of stimulating the spinal cord using electrical impulses, can improve motor function, breathing, and knee motion in people with spinal muscular atrophy types 2 and 3, a study suggests. “Further studies are needed to elucidate the…
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…
Liver issues may be an understudied manifestation of spinal muscular atrophy (SMA), according to a new study suggesting liver involvement in patients warrants greater consideration during disease management. Among a small group of SMA patients, three-quarters showed signs of fat accumulation in the liver, with a few also…
More than half of the children with spinal muscular atrophy (SMA) type 1 who received disease-modifying therapies (DMTs) in their first year of life showed cognitive deficits in a recent study. Cognitive problems were more likely in boys and in those who required assisted ventilation or feeding.
The department of health (DoH) in Abu Dhabi — one of the seven emirates that make up the United Arab Emirates (UAE) — is teaming up with Roche Pharmaceuticals Middle East to advance research that aims to use real-world data to investigate the burden of spinal muscular…
Researchers described what they believe to be the oldest asymptomatic person with genetically confirmed spinal muscular atrophy (SMA) to have ever been reported in a case study. The 60-year-old man, who came in with symptoms of numbness thought to be due to a pinched nerve in the neck,…
Switching to oral Evrysdi (risdiplam) from another disease therapy is safe for spinal muscular atrophy (SMA) patients, with side effects similar to those observed in clinical trials involving patients new to treatment, according to a two-year analysis from the JEWELFISH clinical trial. Exploratory efficacy analyses also suggest…
Spinal muscular atrophy (SMA) patients in the U.S. exhibited high rates of adherence to their prescribed regimen of Evrysdi (risdiplam) over a year, according to a recent analysis of pharmacy prescription fill data. “High adherence to and persistence with [Evrysdi] were observed among patients with SMA over…
I’ve been working with a patient, Michael, who has spinal muscular atrophy. Michael uses a motorized wheelchair, and recently moved to a new chair. Since then, he has experienced increased right hip and tailbone pain off and on with numbness down his right leg as he’s tried to get…
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…
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