Author Archives: Marisa Wexler MS

Behavioral problems such as hyperactivity and aggression are generally more frequent in younger children with fragile X syndrome, becoming less common as they grow through adolescence and journey toward adulthood, researchers report. The study “Change in Behavior Problems from Childhood Through Adolescence for Children with Fragile…

A team led by researchers at Fulcrum Therapeutics has developed new methods to measure the FMRP protein in blood cells. Using these tests, the scientists showed that FMRP levels in blood cells were statistically associated with intelligence quotient (IQ) scores among people with fragile X syndrome. The study,…

Nova Mentis, a Canadian biotechnology company, is teaming up with KGK Science to develop Nova’s portfolio of therapies based on the psychedelic compound psilocybin. The companies are planning to ask Health Canada for the go-ahead to launch a Phase 2 clinical trial in fragile X syndrome that would test…

Zygel, an experimental cannabidiol gel that Zynerba Pharmaceuticals is developing to help normalize behaviors in people with fragile X syndrome, continues to be well tolerated up to a median treatment length of 21 months, according to new data from the open-label extension of the Phase 2/3 CONNECT-FX trial.

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 company developing this treatment candidate, is now looking to begin clinical testing of the medication in…

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…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

As early as 12 months of age, children with fragile X syndrome (FXS) display signs of social anxiety, a study indicates. The study, “Early behavioral and physiological markers of social anxiety in infants with fragile X syndrome,” was published in the Journal of Neurodevelopmental Disorders. Anxiety disorders…

The anticonvulsant carbamazepine can normalize behavior and memory in a mouse model of fragile X syndrome (FXS), suggesting that this medication might be re-purposed to treat people with this disorder, according to a study. The study, “Carbamazepine Restores Neuronal Signaling, Protein Synthesis, and Cognitive Function…