Marisa Wexler, MS,  senior science writer—

People with spinal muscular atrophy (SMA) may show subtle abnormalities in eye movements, but these abnormalities are unlikely to be caused by weak muscles; instead, they likely arise due to changes in the brain, a study found. The study, “Central eye movement abnormalities in patients with SMA types…

Nearly all children with spinal muscular atrophy (SMA) who were started early on Evrysdi (risdiplam) in a clinical trial — as newborns, before the onset of symptoms — are now walking after three years on the approved oral therapy. Further, most of the youngsters also have no need…

The U.S. Food and Drug Administration (FDA) has declined to approve apitegromab as a treatment for spinal muscular atrophy (SMA). The FDA said it could not approve developer Scholar Rock‘s application at this time because of observations identified during a routine general site inspection of Catalent…

Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the end of the year if the U.S. Food and Drug Administration (FDA) approves it next month as an add-on treatment for spinal muscular atrophy (SMA). The company sought…

Treatment with AJ201 was well tolerated and showed signs of improving physical activity in a clinical trial involving people with spinal and bulbar muscular atrophy (SBMA), according to new data announced by developer Annji Pharmaceutical. “I am greatly encouraged by the positive clinical outcomes observed after a relatively…

Injecting medicines into the amniotic fluid — the liquid that surrounds a developing fetus during pregnancy — may be a viable strategy for treating spinal muscular atrophy (SMA) before birth, according to a new study done in animal models. “These data demonstrated that [injecting therapies into amniotic fluid] holds…

A study found that people with spinal muscular atrophy (SMA) have metabolic alterations due to impairments in how their bodies use fatty acids for energy. The results suggested a carbohydrate-rich diet may help normalize metabolism in SMA patients. The study, “Alterations of Fat and Ketone Body Metabolism…

Liver damage may occur as a side effect of the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), but it usually resolves within a few months of treatment, a study showed. The researchers also found that simultaneous treatment with Zolgensma and Spinraza (nusinersen) does not seem to…

A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said. “Evrysdi is the only oral, non-invasive disease-modifying SMA treatment, and the new tablet formulation may provide greater freedom and…

Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to a new analysis. “This study aligns with previously reported findings and suggests that long-term [treatment with Spinraza] offers sustained clinical benefits in adults with SMA,” researchers wrote in the abstract…