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Connecta Therapeutics‘ CTH120 molecule has been granted orphan designation by the European Medicines Agency (EMA) for treating fragile X syndrome, the leading cause of hereditary intellectual disability and autism spectrum disorders. The company said it intends to begin human clinical trials as early as next year to test …

The McQuade Center for Strategic Research and Development (MSRD) will collaborate with Sentinel Oncology for the clinical development of a potential breakthrough treatment for fragile X syndrome (FXS). Under agreement terms, the MSRD, which funds innovative early stage research and development programs, will work with the…

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…