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…
Capsules containing very small doses (microdoses) of psilocybin are ready to be used in a future Nova Mentis clinical trial, with the company one step closer to testing them as a potential treatment for fragile X syndrome. The capsules started being produced per chemistry, manufacturing, and control (CMC)…
People with fragile X syndrome have significantly lower levels of fatty acids — a type of fatty molecule essential for brain health — in the blood than do healthy people, a study showed. These findings add to data from previous studies reporting lower-than-normal cholesterol levels in this patient population.
An enzyme called matrix metalloproteinase 9 (MMP-9) is found at higher levels in the blood of people with fragile X syndrome than healthy people and may correlate with more aberrant behavior and anxiety-related symptoms, a study reported. Researchers determined total levels of MMP-9 and of its active form, but…
A new tool that uses artificial intelligence (AI) to analyze healthcare records may aid in the early diagnosis of fragile X syndrome, a new study reports. “By incorporating a combination of co-occurring conditions, an AI-assisted pre-screening tool was developed and validated to identify potential cases at least 5 years…
AKT, an enzyme that is involved in multiple cellular processes, is overactivated in neural stem cells that lack the FMRP protein, as is the case in fragile X, leading to the stimulation of a cellular quality control pathway called NMD, according to a new study. Blocking AKT in these…
A new fund at the University of California, Davis (UC Davis) MIND Institute seeks to speed up turning new research discoveries into therapeutic interventions for people with fragile X syndrome and other developmental conditions. The Translational Research Impact Fund was established by a posthumous donation from Leon Goldstein, who…
Get regular updates to your inbox.