Author Archives: Marisa Wexler MS

Without treatment, most people with spinal muscular atrophy (SMA) who have four copies of the “backup” SMN2 gene will start developing symptoms in early childhood, a study indicates. “The early onset of symptoms in our cohort, coupled with the expected wide clinical variability, strongly supports the characterization of…

In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), subsequent treatment with Spinraza (nusinersen) can improve motor function and may reduce nerve damage. That’s according to interim data from the Phase 4 RESPOND study…

The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) didn’t cause any unexpected safety issues among children with spinal muscular atrophy (SMA) who weighed up to 21 kg (about 46 pounds) in a Phase 3b clinical trial, and most had stable or improved motor function a year after treatment.

Children with spinal muscular atrophy (SMA), even those with substantial physical disability, tend to report that their quality of life is similar to other children their age, a small study from Germany reports. “The results from this pilot study show that German children with SMA, despite significant physical disability,…

A Phase 1b/2a clinical trial testing the experimental therapy AJ201 in people with spinal and bulbar muscular atrophy (SBMA) has finished enrolling patients and topline data are expected in the first half of 2024. The milestone was announced by Avenue Therapeutics, which last year acquired rights to…

The gene therapy Zolgensma (onasemnogene abeparvovec) did not cause any serious long-term safety problems when used in children with type 1 spinal muscular atrophy (SMA) in a real-world study from the U.K., and most treated patients experienced improvements in motor function. “This is one of the largest studies…

Treatment with Spinraza (nusinersen) led to modest improvements in motor function for most children with type 1 spinal muscular atrophy (SMA) who were on permanent ventilation in a Korean study. The results are particularly noteworthy because Spinraza is approved in South Korea only for those patients who are…

More work is needed to expand access to the gene therapy Zolgensma (onasemnogene abeparvovec) for people with spinal muscular atrophy (SMA) living outside of wealthy countries like the U.S. or Australia. That’s the argument made by researchers in the paper, “Gene therapy-based strategies for spinal muscular…

Adults with spinal muscular atrophy (SMA) type 3 showed deficits in certain areas of cognition, particularly executive function or the ability to regulate one’s own actions, according to a recent study in Chinese patients. Data from the small analysis suggest that these issues tend to be more pronounced in…

Treatment with Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec) led to increases in the electrical activity of motor neurons — the specialized nerve cells that control voluntary movement — for two young children with type 1 spinal muscular atrophy (SMA), according to a new report. Findings from tests…