Survival outcomes in spinal muscular atrophy (SMA) are heavily influenced by age of onset, even among patients who are classified as having the same type of SMA, according to an analysis of more than three decades’ worth of data at a center in Thailand. In fact, data suggest…

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 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…

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…

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…

Mom and I always bring another person or two to my specialist appointments at the SMA clinic. It’s out of state — about three hours from our home — so it’s easier when we have extra helpers throughout the long journey. It’s also nice to have company.

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…

Twenty-six children were diagnosed with spinal muscular atrophy (SMA) in Russia through a 2022 pilot program, out of nearly 203,000 newborns who were screened for the rare genetic disease. That’s according to a study, “Epidemiology of Spinal Muscular Atrophy Based on the Results of a Large-Scale…