Zygel, an experimental cannabidiol (CBD) gel, may lessen the extent of behavioral abnormalities in children with fragile X syndrome who have more severe disease, new clinical trial data suggest. The data were presented in a poster, titled “ZYN002 Cannabidiol Transdermal Gel in…
The National Institutes of Health (NIH) is awarding multi-year grants to three centers conducting collaborative research into fragile X syndrome: Baylor College of Medicine, Emory University, and Cincinnati Children’s Hospital Medical Center. Collectively, the centers will receive $25 million over the next five years, with…
Clinical improvements among participants receiving a placebo — known as the placebo effect — are common in clinical trials of fragile X syndrome, a study conducted by scientists across the U.S. suggests. The findings indicate that better trial designs, including different efficacy measurements, may be necessary in future…
The experimental cannabidiol (CBD) gel Zygel eased behavioral problems in children with fragile X syndrome and benefited those with greater disease severity according to their caregivers, new clinical trial data show. The data were presented virtually at the 17th NFXF International Fragile X Conference Research Roundup by…
The common type 2 diabetes medication metformin eased the excessive production of proteins in nerve cells that were engineered in a lab to have the underlying defect seen in people with fragile X syndrome, a study reported. These findings support further development of metformin as a treatment for fragile…
Zynerba Pharmaceuticals‘ Zygel failed to significantly ease behavioral symptoms in the overall group of children with fragile X syndrome (FXS) who took part in the CONNECT-FX trial, top-line results show. However, significant benefits were detected in participants with more severe symptoms, indicating that the experimental…
Top-line results from the CONNECT-FX trial testing the cannabidiol (CBD) gel Zygel as a potential treatment for behavioral symptoms in children and teenagers with fragile X syndrome are expected by late June. Should findings be positive, Zynerba Pharmaceuticals plans to request a meeting with the U.S. Food…
A procedure for measuring language skills, called expressive language sampling (ELS), could be used to assess treatment outcomes in people with fragile X syndrome and other conditions characterized by intellectual disability, a new study suggests. The study, “Expressive language sampling as a source of outcome measures for…
Using a genetic technique called triplet-primed polymerase chain reaction (TP-PCR) could enable practical and inexpensive diagnosis of fragile X syndrome, a study suggests. The study, “Repeat expansion and methylation-sensitive triplet-primed polymerase chain reaction for fragile X mental retardation 1 gene screening in institutionalised intellectually disabled individuals,”…
The U.S. Food and Drug Administration (FDA) has authorized the marketing of Asuragen‘s AmplideX Fragile X Dx and Carrier Screen Kit, a first genetic test to help diagnose fragile X syndrome and determine the likelihood of having a child with the condition. “This novel diagnostic provides doctors and their…
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