Marisa Wexler, MS, senior science writer —

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…

In an effort to both increase accuracy and remove offensive language in the name of FMR1 — the gene at the root of fragile X syndrome — the European Fragile X Network (EFXN) led a push to rename it Fragile X Messenger Ribonucleoprotein 1. The name change now…

In people with fragile X-associated tremor/ataxia syndrome (FXTAS), a condition related to fragile X syndrome that develops late in life, measures of dexterity and memory tend to worsen markedly over time, a new study shows. The results support the development of preventive treatments for FXTAS, according to its researchers.

Unusually low amounts of cholesterol in cellular structures called lipid rafts may contribute to behavioral differences seen in people with fragile X syndrome, a new study indicates. “The correlation between [lipid rafts] cholesterol and the clinical profile showed a significant association with autistic traits,” the researchers wrote. The study, “…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

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.