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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 capable…

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 showing…

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. “Given…

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 atrophy…

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, was…

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 that…

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 the…

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 a…

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 effects…

Jerry R. Mendell, MD, has been named to the inaugural TIME100 Health list for his decades-long contributions to the treatment of neuromuscular diseases — such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) — and the advancement of gene therapy. The global media brand’s list…