Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has given Alcyone Therapeutics the go-ahead to start a clinical trial testing ThecaFlex DRx, its implantable system for repeat use of Spinraza (nusinersen) to treat people with spinal muscular atrophy (SMA). The pivotal trial, called PIERRE (NCT05866419), will be conducted…

Adults with spinal muscular atrophy (SMA) who start treatment with Spinraza (nusinersen) tend to see the most dramatic early improvement in muscle strength in the shoulders and upper arms, a new study suggests. The treatment also tended to improve thumb strength, which researchers note may be important for…

Children with spinal muscular atrophy (SMA) who are treated with Zolgensma (onasemnogene abeparvovec-xioi) tend to have more dramatic gains in motor function when it’s given earlier in the course of disease, a new study reports. The findings suggest the best improvements are seen among babies treated in the…

A second-generation gene therapy, administered directly into the brain, led to faster and more pronounced improvements on motor function in a mouse model of spinal muscular atrophy (SMA) compared with a benchmark vector similar to the approved gene therapy Zolgensma (onasemnogene abeparvovec-xioi). That’s according to new data announced…

The side effects that some patients experience on Evrysdi (risdiplam), an approved treatment for spinal muscular atrophy (SMA), may be due to how the therapy alters the activity of thousands of genes when given at high doses, according to a new study in cell models. Findings also suggest…

Treatment with Evrysdi (risdiplam) in spinal muscular atrophy (SMA) patients who previously received other therapies was found to safely stabilize motor function and lead to slight improvements in arm function, new clinical trial data show. These results occurred “irrespective of previous treatment,” the researchers wrote, adding that “no…

In adults with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen) in a clinical trial was tolerated well and led to improvements in arm function and self-rated health. These findings add to data from several studies that have examined the safety and efficacy of Spinraza in older patients…

Twin girls with type 0 spinal muscular atrophy (SMA) — the most severe form of the disease, which usually leads to death in the first months of life — are alive nearly two years after receiving early treatment with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), according to researchers.

Children and adolescents in New Zealand with spinal muscular atrophy (SMA) will be able to access Evrysdi (risdiplan) through the country’s publicly funded healthcare system, starting in May. The decision by New Zealand’s Pharmaceutical Management Agency, known as Pharmac, means eligible patients will now have access to two…

The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA) effectively, especially when it’s given in the first months of life, a review paper shows. “There is substantial evidence of improved outcomes when Zolgensma is administered early to children under…