News

The Muscular Dystrophy Association (MDA) has opened the MDA Gene Therapy Support Network — a new program it’s dubbed GTx — to aid and educate families living with spinal muscular atrophy (SMA) or other neuromuscular diseases (NMDs) who may be considering a gene therapy as part of…

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…

Alterations to the composition of intestinal microbes — called the gut microbiome — in spinal muscular atrophy (SMA) patients may contribute to certain metabolic disorders that are commonly seen in patients, according to a recent study. Scientists identified a number of altered gut bacteria in children with SMA relative…

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…

In real-world settings, the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is well tolerated and improves motor function outcomes in children with spinal muscular atrophy (SMA) — especially when given at ages younger than 8 months — a study in Israel found. Treatment with Zolgensma also was found to…

Boosting the production of the growth factor protein NRG1-III improved motor nerve fiber development in the early stages in a mouse model of spinal muscular atrophy (SMA), a study showed. NRG1-III was unable to prevent nerve fibers from degenerating or improve motor function or survival in older mice, however.

The loss of SMN protein in spinal muscular atrophy (SMA) may lead to disease features by causing a deficiency in another enzyme called ALDH1A2, which is essential for the maturation and expansion of motor neurons, the cells that are lost in SMA, according to a new study. The findings…

The MiR34 family of microRNAs represents a set of promising biomarkers to assess therapeutic response in spinal muscular atrophy (SMA), a study proposes. MicroRNA levels in the cerebrospinal fluid (CSF), the liquid surrounding the brain and spinal cord, before and during therapy, predicted and correlated with improved motor function…

Treatment with Spinraza (nusinersen) for two years led to improvements in apnea and oxygen saturation during sleep in children with spinal muscular atrophy (SMA), a real-world study in Australia reported. The treatment also contributed to ceasing ventilation support in some patients with less severe disease, and stabilized lung…

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…