News

Children with Fragile X syndrome (FXS) share some traits with those with autism spectrum disorder (ASD), but exhibit several differences in terms of brain’s traffic patterns, according to an MRI study. Associations between certain brain traffic patterns, which reflect different connections between brain regions that actively work together, and scores…

SPG601, an oral treatment candidate now in clinical testing, has been granted orphan drug status for fragile X syndrome in the European Union. This designation, awarded by the European Medicines Agency (EMA), is meant to incentivize companies developing treatments for rare diseases — those affecting fewer than 5 of every…

Supporters worldwide are gearing up to celebrate Fragile X Awareness Month, an initiative held each July to boost recognition of fragile X syndrome and advocate for funding to improve the lives of people living with the rare disease. The monthlong event is to be celebrated alongside the fifth annual…

KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and…

Long-term, daily treatment with the investigational cannabidiol (CBD) gel ZYN002 — also known as Zygel — led to clinically meaningful reductions in irritability and other behavioral symptoms in people with fragile X syndrome. That’s according to up to three years of data from an ongoing open-label extension…

KER-0193, Kaerus Bioscience’s oral treatment candidate for fragile X syndrome, was safe and well tolerated, and showed predictable pharmacological properties, in healthy volunteers, according to new data from a Phase 1 clinical trial. The therapy was also associated with changes in brain activity in regions known to be…

A single dose of Spinogenix’s investigational oral therapy SPG601 led to significant reductions in a measure of abnormal brain activity among men with fragile X syndrome, according to top-line results from a Phase 2a trial. Announced by Spinogenix in a company press release, the results from the…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Spinogenix‘s experimental oral therapy SPG601 to treat fragile X syndrome (FXS). The designation aims to speed the development and review of new medicines that have the potential to improve medical care for serious conditions. It…

Researchers in Kansas and California will investigate people with the FMR1 premutation who are at risk of developing fragile X-associated tremor/ataxia syndrome (FXTAS), a condition related to fragile X syndrome that develops late in life. The five-year project, supported by a $3.1-million grant from the National Institutes of Health, will…

  A Phase 2 clinical trial to test MRM-3379, an experimental oral treatment for fragile X syndrome, is expected to launch in 2025. Mirum Pharmaceuticals announced the plan in a company press release reporting financial results and business updates. It didn’t provide details on the trial’s design…