Author Archives: Lila Levinson, PhD

What is Spinraza for SMA? Spinraza (nusinersen) is an injection therapy widely approved for treating adults and children with spinal muscular atrophy (SMA). It was the first therapy approved to target the underlying causes of SMA. Mutations in the SMN1 gene, which contains instructions for cells to produce…

The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results…

Up to three years of treatment with Evrysdi (risdiplam) appears safe and was associated with presymptomatic children with spinal muscular atrophy (SMA) reaching and maintaining key developmental milestones, according to Phase 2 study…

A high-dose regimen of Spinraza (nusinersen) that is up for regulatory approval in the U.S. may help stabilize or improve motor function for people with spinal muscular atrophy (SMA), pooled results from two clinical trials…

Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in children…

Genetic screening for spinal muscular atrophy (SMA) in newborns may allow treatment to begin earlier, which could help support motor development, a study suggests. This “adds to the evidence that [newborn screening] for SMA is…

Apitegromab, a muscle-strengthening therapy for spinal muscular atrophy (SMA), helped improve motor function in a new analysis of Phase 3 trial data, with the greatest gains seen in people who started treatment earlier. In…

Modifications that help certain genetic information travel to specific parts of nerve cells can ease motor symptoms and help normalize communication between nerves and muscles in a mouse model of spinal muscular atrophy (SMA), a study reported. SMA…

Using a specially designed back brace in addition to taking part in physical therapy may work best to reduce abnormal spine curvature, or scoliosis, in children with spinal muscular atrophy (SMA) type 1, a…

A technique to estimate the health of nerve cells that control movement may help predict how strongly some individuals will respond to the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a study reports. This…