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Note: This story was updated Aug. 12, 2022, to reflect that blarcamesine is an experimental activator.  Treatment with Anavex2-73 (blarcamesine) safely and significantly eased behavioral symptoms of fragile X syndrome (FXS), and normalized some aspects of brain chemistry, in a preclinical study in mice. Anavex Life Sciences, the…

A Rush University Medical Center scientist will use a $4 million “cooperative agreement” from the Centers for Disease Control and Prevention (CDC) to better define the natural history of fragile X syndrome (FXS) and determine ways to improve the lives of patients and their families. The five-year study,…

Nova Mentis is seeking orphan drug designation from the European Medicines Agency (EMA) for psilocybin, its investigational therapy for fragile X syndrome. Psilocybin, found naturally in certain mushrooms — popularly called “magic mushrooms” — is most known for its hallucinogenic, anxiolytic, and psychoactive effects. However, Nova officials…

Synaptogenix and Nemours A.I. DuPont Hospital for Children have signed an agreement to collaborate on a clinical trial to test the investigational medication bryostatin-1 in people with fragile X syndrome (FXS). “We are excited to participate in a potentially breakthrough therapeutic drug strategy to address this huge…

A $90,000 grant from the FRAXA Research Foundation will fund a preclinical study that aims to assess the potential benefits of a diet that is high in fat and low in carbohydrates for fragile X syndrome using mice and cell models. A high fat/low carbohydrate diet is known to be…

In a rural village in Cameroon, in West Africa, social stigma against those with intellectual disabilities such as fragile X syndrome exists in an uneasy relationship with a deeply ingrained tradition of deference to social status, according to a new ethnographic study. A group of researchers studying this phenomenon…

The FRAXA Research Foundation is supporting a project to identify available medicines that might be repurposed to treat major depressive disorder (MDD) in people with fragile X syndrome. Depression occurs frequently among people with autism spectrum disorders (ASD), of which fragile X is the most common genetic cause.

The U.S. Food and Drug Administration (FDA) has given positive feedback to Actinogen’s pre-investigational new drug (pre-IND) submission regarding Xanamem, its experimental oral therapy for fragile X syndrome. In a written response following a pre-IND meeting with Actinogen, the FDA agreed the data gathered and the proposed trial design…

From posting yard signs to swapping out exterior lighting, people across the U.S. are poised to mark Fragile X Awareness Month, observed each July. A global day — July 22 — is also set aside, with illuminations worldwide helping to call attention to the genetic disorder. Awareness and education…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…