Marisa Wexler, MS, senior science writer —

Screening of potential participants has begun in a Phase 2a trial that will test Nova Mentis Life Science’s NM-1001, a microdose of the psychedelic molecule psilocybin, in adults with fragile X syndrome. The study (NCT05832255), which is being conducted in a partnership with KGK Science,…

PureTech Health is planning a Phase 2 clinical trial to test its lead therapeutic candidate LYT-300 — an oral formulation of allopregnanolone — in people with fragile X-associated tremor/ataxia syndrome (FXTAS). The company has been awarded a grant of up to $11.4 million from the U.S. Department of Defense…

Attention-deficit/hyperactivity disorder (ADHD) is more common among female carriers of a fragile X premutation who have more cell-to-cell variation in the number of mutational CCG repeats in the premutation, a new study reveals. Indeed, according to researchers, “the degree of [genetic] instability (expansion) was significantly higher … in participants…

KGK Science will launch a Phase 2a trial in the coming months to test NM-1001, a psilocybin microdose therapy — meaning one that contains low doses of the hallucinogenic compound — in people with fragile X syndrome. The company has received a needed exemption from the Canadian government…

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…

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…

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…

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…

Treatment with Anavex2-73 (blarcamesine), an experimental therapy that Anavex Life Sciences is developing to treat fragile X syndrome led to certain signaling pathways in immune cells normalizing in mouse model experiments. Analyzing these immune signaling pathways could help track the effect of the investigational medication in future clinical…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Neuronascent‘s investigational therapy NNI-351 for the treatment of fragile X syndrome (FXS). “With vast unmet need for novel therapies that can directly address pediatric FXS behavioral deficits, such as to improve education and social…