News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Ketarx (racemic ketamine) for treating Rett Syndrome. The designation is awarded to products that show promise to prevent, diagnose, or treat rare diseases. Incentives for developers include seven years of market exclusivity after approval, potential…

The Rett Syndrome Research Trust (RSRT) is supporting ongoing studies of HRP-12975, an investigational therapy for Rett syndrome, using mouse models of the disease. HRP-12975, being developed by Herophilus, is reported to be the first small molecule therapy with the potential to reverse MECP2 gene deficiency, the…

The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s investigational new drug application for NGN-401, an experimental gene therapy for Rett syndrome. This paves the way for the therapy to be tested in a planned Phase 1/2 clinical trial in girls with Rett syndrome, to begin later…

Scientists may have discovered promising treatments for Rett syndrome in two small molecules, called KW-2449 and VPA, each seen to ease nerve cell deficits — thereby potentially improving the flow of information between neurons — in a lab model of the disorder. “These findings suggest that KW-2449 and VPA…

Treatment with the diabetes medication metformin for four months led to cognitive improvements and restored brain energy production in a mouse model of Rett syndrome, a study found. Notably, the therapy’s benefits were observed when administered in a relatively advanced stage of disease — one in which the…

Girls with Rett syndrome caused by MECP2 gene mutations have increased deep sleep and reduced rapid eye movement (REM) sleep, a study shows.  The sleep patterns are generally similar across the patients’ genetic and clinical, or disease severity, characteristics. However, increased hand disability was associated with a…

Nerve cells derived from Rett syndrome patients showed alterations in structure, function, and network connectivity that depend on the severity of MECP2 genetic changes that cause the condition, a study revealed. Nerve cells from a rare atypical Rett patient with mild disease and preserved speech had similar core structural…

As part of its Project Seva, Vezbi Community Super App is supporting the International Rett Syndrome Foundation (IRSF) through a $500,000 equity donation and the creation of tailored microapps for the Rett syndrome community. The community-focused Super App was created to consolidate and organize all life aspects…

A new mutation has been found in the MECP2 gene — associated with most Rett syndrome cases — in a toddler with clinical features suggestive of congenital variant-like Rett syndrome, one of the most severe atypical forms of the disease. The child had hypercapnia, or high carbon dioxide levels…

A study on the brain’s molecular anomalies in Rett Syndrome received $100,000 in funding from the Canadian Institutes of Health Research (CIHR). The research will be conducted for a year by the team of Mojgan Rastegar, PhD, at the University of Manitoba, Canada. It’s one of six studies from…