News

Cell growth and signaling, as well as cholesterol production, are significantly altered in lab-grown astrocytes — the star-shaped brain cells that provide support to neurons — derived from people with fragile X syndrome, a study shows. The patient-derived cells also had altered levels of a number of proteins that…

A pilot study in the U.S. shows that repeated sampling of hair follicles in people with fragile X syndrome, collected both at home and clinics, can be used to measure the levels of FMRP protein — whose production is impaired in fragile X patients. In addition, measuring FMRP levels using…

Treatment with a small molecule called pireperone rescued cognitive and behavioral impairments in a mouse model of fragile X syndrome, making it a potential therapy candidate for the rare genetic disorder, a study reported. Pireperone’s beneficial effects were likely associated with a significant increase in the activity of the…

Caregivers can accurately recognize signs of anxiety in people with fragile X syndrome whose ability to articulate their emotional state is limited, a new study shows. “While most people with FXS [fragile X syndrome] cannot reliably describe their internal body or emotional states to support self-reported assessment procedures, the…

A tripledemic — high rates of respiratory syncytial virus, influenza, and COVID-19 — is slowing enrollment in the Phase 3 clinical trial testing Zygel, Zynerba Pharmaceuticals’ experimental cannabidiol gel, in children and adolescents with fragile X syndrome. These environmental factors are causing unexpectedly higher cancellations of the initial…

The NIH Toolbox Cognition Battery (NIHTB-CB) test accurately detects developmental changes in cognitive function among young people with intellectual disabilities, including those with fragile X syndrome, a study reports. Matching, or even exceeding, the changes detected in a standard IQ test over two years, the NIHTB-CB is a…

KGK Science is requesting permission from Health Canada to conduct a Phase 2a clinical trial evaluating a microdose of the psychedelic compound psilocybin in people with fragile X syndrome. This first in-human trial seeks to investigate the safety of this psilocybin microdose, called NM-1001, as well as its…

KGK Science, a wholly owned subsidiary of Wellbeing Digital Sciences, has entered into an agreement with Nova Mentis Life Science to conduct a Phase 2a clinical trial testing the efficacy of psilocybin as a potential treatment for fragile X syndrome. The open-label trial will be the…

Many children with fragile X syndrome have had poorer academic performance, worsening mental health problems, and more difficulties with social interaction after the COVID-19 pandemic, according to a survey of parents. Problems were generally less severe among children who continued to be schooled in person and parents tended to…

Anavex2-73 (blarcamesine), an experimental small molecule being developed by Anavex Life Sciences, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) as a potential treatment of fragile X syndrome. This designation is given to investigational treatments for people with rare diseases, defined…