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…
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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…
The amount of neurofilament proteins found in the cerebrospinal fluid, the liquid surrounding the brain and spinal cord, may help doctors better distinguish between adult-onset spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS), a study found. The two conditions are marked by damage to motor neurons, the…
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.
