A group of European experts has published updated recommendations for using the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in people with spinal muscular atrophy (SMA) that includes new cautions with older, heavier patients. “2024 Update: European Consensus Statement on Gene Therapy for Spinal Muscular Atrophy,” was…
News
More than 90% of children with spinal muscular atrophy (SMA) type 1 who started taking Evrysdi (risdiplam) as babies are still alive and achieving motor milestones that would be unattainable without treatment, according to final, five-year data from the FIREFISH clinical trial. The findings were presented at the…
A method that uses laser light to get information from inside the body in a noninvasive way may help monitor how spinal muscular atrophy (SMA) is progressing in real time and how children are responding to therapy, a study suggests. Signals from optoacoustic imaging, which can picture how well…
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 reasons…
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…
