José Lopes, PhD,  managing science editor—

Lower levels of the FMRP protein in neural stem cells delay their conversion into neurons during brain development by disrupting the transport of RNA molecules, new research shows. Scientists say those decreased FMRP levels are likely linked to impaired learning and behavior in fragile X syndrome. These findings…

Treatment with the Alzheimer’s therapy donepezil reduced brain activation in response to a gaze stimulus, but did not directly improve cognition or correct abnormal behavior in patients with fragile X syndrome (FXS), according to a 12-week Phase 2 trial. The study, “Brain circuitry, behavior, and cognition:…

Anxiety, learning difficulties, and behavior problems are the main areas of concern in patients with fragile X syndrome (FXS), according to a survey that included patients, family members or caregivers, and healthcare professionals. Survey results, which also showed that anxiety is viewed as the top treatment priority, were reported in…

A greater ability to generate words, phrases and sentences correlates with lower autism spectrum disorder (ASD) symptom severity in adolescent and young adult males with fragile X syndrome (FXS), according to a study. The research, “ASD Comorbidity in Fragile X Syndrome: Symptom Profile and Predictors of Symptom Severity in Adolescent and Young Adult…

Mavoglurant is safe and well-tolerated in the long-term to treat adolescents and adults who have fragile X symptoms. The investigational compound also is associated with positive efficacy results, according to results from two clinical studies. The research with those findings, “Mavoglurant in Fragile X Syndrome: Results of…

Altered calcium levels and poorly regulated activity in specific calcium channels in nerve cells could underlie fragile X syndrome (FXS) and be used as a diagnostic and treatment biomarker, according to a new study. The research, “New Insights Into the Role of Cav2 Protein Family in Calcium…

High levels of a pro-inflammatory molecule called interleukin (IL)-6 in the brain could be key to abnormal neuronal communication in fragile X syndrome, a mouse study reports. The study, “Regulation of IL-6 Secretion by Astrocytes via TLR4 in the Fragile X Mouse Model,” appeared in the…

  A University of Calgary team attempting to reverse symptoms of fragile X syndrome (FXS) in mice was awarded a $100,000 grant by the FRAXA Research Foundation and the Fragile X Research Foundation of Canada. Fragile X syndrome, the most common genetic cause of autism, is caused by lack…

The U.S. Food and Drug Administration (FDA) recently granted fast track designation to Ovid Therapeutics’ Fragile X syndrome (FXS) treatment candidate OV101 (gaboxadol). The designation accelerates the development and regulatory review of investigational medications for the treatment of serious conditions and that fill an unmet medical need.

Zynerba Pharmaceuticals will start a clinical trial of its cannabis-based Fragile X syndrome treatment  ZYN002 in mid-2018. The U.S. Food and Drug Administration gave the company a green light for the trial at a recent meeting. Zynerba expects the results to support the filing of a New Drug Application for…