Washington University School of Medicine in St. Louis is one of only ten sites in the world participating in a pioneering clinical trial evaluating TSHA-102, an investigational gene therapy for the treatment of Rett syndrome, a severe neurodevelopmental disorder primarily affecting girls. The REVEAL Phase 1/2 clinical trial represents a significant advancement in the quest […]
Studies have shown that Black children with serious illnesses are less likely than white children to obtain crucial genetic testing necessary to guide treatment decisions, but the reasons for this disparity have not been fully understood. A new study from Washington University School of Medicine in St. Louis focused on children with neurological conditions finds […]
In the past two years, the Food and Drug Administration has approved two novel Alzheimer’s therapies, based on data from clinical trials showing that both drugs slowed the progression of the disease. But while the approvals of lecanemab and donanemab, both antibody therapies that clear plaque-causing amyloid proteins from the brain, were greeted with enthusiasm […]
Remarkably, Doug Whitney, 75, has escaped genetic destiny. Like many members of his family, Whitney inherited a rare genetic mutation that all but guarantees he would develop early-onset Alzheimer’s disease. But Whitney, whose relatives first showed symptoms of cognitive decline in their early 50s, remains mentally sharp with no signs of the devastating disease, and […]
The first participants in an international clinical trial aimed at preventing Alzheimer’s disease in young adults at high risk of the disease have been enrolled. The trial, led by Washington University School of Medicine in St. Louis, aims to determine whether stopping the early molecular changes that lead to symptomatic Alzheimer’s disease can prevent the […]
Individuals with sickle cell disease – a chronic illness where misshapen, sticky blood cells clump together, reducing oxygen delivery to organs – are at a higher risk for stroke and resulting cognitive disability. But even in the absence of stroke, many such patients struggle with remembering, focusing, learning and problem solving, among other cognitive problems, […]
For decades, the general consensus among neurologists and neuroscientists has been that ALS treatments would only be able to slow down or stop further progression of the disease. Function that had already been lost would probably not be recovered. But now, evidence is emerging that not only is functional improvement possible, it’s happening for some people […]
As a leading researcher of rare diseases that affect children’s brains, Jonathan D. Cooper, PhD, thought little about the gastrointestinal (GI) system. That is, until the parents of children with a condition that Cooper studies urged him to investigate why debilitating digestive issues troubled their kids, who suffer from an incurable and fatal neurodegenerative brain condition […]
The gas xenon, like the other noble, or inert, gases, is known for doing very little. The class of elements, because of its molecular structure, don’t typically interact with many chemicals. But a new mouse study shows one possible use case for xenon — as a treatment for Alzheimer’s disease. The paper, published Wednesday in Science Translational […]
When studying substance use disorders, scientists had thought some of the effects on the brain could stem from use of the substances themselves: People start drinking alcohol in early teens, that alcohol has a neurotoxic effect on the developing brain that begets more alcohol drinking, and a similar dynamic occurs with other substances. But research […]