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A five-year, $2.4 million grant will fund a new study of grammar skills and executive functions in preschool and school-age children with fragile X syndrome. The grant, awarded by National Institute on Deafness and Other Communication Disorders, part of the National Institutes of Health, will be…

A candidate therapy for fragile X syndrome — developed using Quris’ bio-artificial intelligence clinical prediction platform — will enter clinical trials in 2022, the company announced. Quris is using the power of artificial intelligence (AI)  combined with a “patients-on-a-chip” platform to predict the safety and potential effectiveness of therapeutic…

Nova Mentis has filed an application for orphan drug designation with the U.S. Food and Drug Administration (FDA) for psilocybin, its investigational psychedelic therapy for fragile X syndrome. Recently, psilocybin received orphan drug designation from the European Medicines Agency (EMA), which offers financial incentives for potential rare disease treatments. Nova also…

A Phase 2a clinical trial of HLX-0201 in treating behavioral symptoms in people with fragile X syndrome was given U.S. Food and Drug Administration (FDA) approval to open, the therapy’s developer, Healx, announced. Recruitment for the trial (NCT04823052) should begin in the coming months, the company stated in a…

Many COVID-19 clinics are large, crowded, and on the boisterous side, which can be overwhelming for people with fragile X syndrome and other neurodevelopmental disorders needing to go there for vaccination. Such concerns led the UC Davis MIND Institute in Sacramento, California, to open a custom-designed clinic for vaccine administration. The…

Zynerba Pharmaceuticals is recruiting children and adolescents with fragile X syndrome for a large-scale clinical trial testing the safety and efficacy of the cannabidiol (CBD) gel Zygel for treating behavioral problems. The Phase 3 trial, called RECONNECT, is enrolling participants ages 3—17 at 26 sites across the U.S., Australia,…

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,…