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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…
Tetra Therapeutics has announced the opening of its first site for a Phase 3 clinical trial assessing the safety and effectiveness of BPN14770 (zatolmilast), its investigational oral therapy for fragile X syndrome. The Suburban Research Associates, a clinical site in Pennsylvania, is now recruiting male…
A month of treatment with retinoic acid — a naturally occurring byproduct of vitamin A — was able to restore certain social behaviors in a mouse model of fragile X syndrome, according to a recent study. The molecule likely restored the behaviors by normalizing levels of certain genes in…
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