Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA), slowed neurodegeneration, and improved motor function, in children with the genetic disease. The data also demonstrated that four of eight SMA children treated for at least one year attained new…
A real-world study of Spinraza (nusinersen) use in Switzerland among individuals with spinal muscular atrophy (SMA) found that, while patients tended to experience motor function benefits mostly in their first year of treatment, many continued to see gains or disease stabilization for four years. Spinraza treatment had the…
The use of salbutamol may safely improve upper limb strength, as well as provide other benefits, in people with spinal muscular atrophy (SMA), particularly among young children, according to data from a new meta-analysis — though the researchers noted that controlled studies are needed to confirm these findings. Two…
Muscle-related body composition measures improved in children with spinal muscular atrophy (SMA) types 2 and 3 who were treated with Spinraza (nusinersen), and the improvements correlated with better motor function, a study found. “Indicators of muscle mass increased in children with SMA types 2 and 3 as treatment…
The case of a woman with spinal muscular atrophy (SMA) type 2 who developed an abnormal accumulation of molecules called ketone bodies was described in a recent case report in the U.S. Called ketoacidosis, this occurs when fats are broken down to obtain energy, resulting in ketone bodies being…
It is with deep sadness and heavy hearts that we announce the passing of our beloved friend, coworker, and advocate, Michael Morale. Michael died in his home in Irving, Texas, on June 7 at the age of 59. I first encountered Michael through his personal YouTube channel in 2018.
Most adults and adolescents with spinal muscular atrophy (SMA) experience intermittent pain, with more than half having pain episodes at least once a week, a study reports. This pain was generally mild to moderate and affected the hips or spine. People with more depressive symptoms tended to have more…
The European Commission (EC) has approved a tablet formulation of spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam). “The new Evrysdi tablet with its flexible administration represents progress toward more versatile SMA disease management,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche,…
A quantitative MRI scan, which shows detailed images of muscles, can be used to track changes seen with Spinraza (nusinersen) treatment in children with spinal muscular atrophy (SMA), according to a new study from China. SMA children started on the approved therapy at a younger age also were found to…
Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized, ride-on cars may boost their confidence, help them connect with others, and make them feel more included, a small study found. “Powered mobility not only facilitates movement but also promotes…
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