News

Nova Mentis has launched an observational study examining the interconnected roles that gut microbes, serotonin, and the immune system play in the gut and brain development of people with autism spectrum disorders (ASDs), including fragile X syndrome. The study (NCT04869930) aims to develop a set of tools — a…

Applications are being accepted for an innovative new four-year college program at the University of California, Davis (UC Davis) for students with intellectual disabilities such as fragile X syndrome. The program is said to be the first of its kind in California and is a collaboration of…

An artificial intelligence (AI) model based only on the occurrence of health conditions previously and newly associated with fragile X syndrome accurately identified fragile X patients five years earlier than diagnosis, according to a study based on nearly 5,600 people in Wisconsin. Among early signs of fragile X were not…

A mouse model of fragile X syndrome has higher body weight and distinct metabolic changes relative to healthy mice and mouse models of two other neurodevelopmental disorders, a study shows. The findings highlight not only that each mouse model exhibits a unique, sex-specific metabolic signature, but also that problems…

Zynerba Pharmaceuticals reported receiving advice from the U.S. Food and Drug Administration (FDA) on the design of an upcoming Phase 3 clinical trial meant to confirm previous trial findings supporting  Zygel as a cannabidiol treatment in a specific subset of fragile X syndrome patients. The new trial, called…

Zynerba Pharmaceuticals’ experimental cannabidiol (CBD) gel Zygel results in statistically significant and clinically meaningful reductions in behavioral symptoms in children and adolescents with fragile X syndrome (FXS) and a fully methylated FMR1 gene, according to data from the Phase 2/3 CONNECT-FX trial. DNA methylation is a biochemical modification that…

Nova Mentis is creating a North American data bank of people with autism spectrum disorders (ASD), which it will use for a study evaluating serotonin levels in ASD patients, including those with fragile X syndrome. The company expects that serotonin levels in various human tissues — including blood,…

Tetra Therapeutics’ investigational oral therapy BPN14770 safely and effectively improves cognitive function, particularly language-related domains, and daily functioning in men with fragile X syndrome, according to data from a Phase 2 clinical trial. “These results offer hope for patients with fragile X syndrome and their families,” Elizabeth…

Abnormal protein production associated with fragile X syndrome was coupled with altered cell growth that favored fast-growing cell types over those that become nerve cells during early development, a study has shown.  Blocking a signaling pathway known as PI3K corrected both excess protein production and…

Better access to information about fragile X-associated ataxia/tremor syndrome (FXTAS) could help improve the quality of life for men carrying fragile X syndrome premutations, according to the results of a small survey. The study, “Men with an FMR1 premutation and their health education needs,” was published in…