Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration (FDA) and Zynerba Pharmaceuticals will meet later this year to discuss the results of the CONNECT-FX trial and next steps regarding a potential regulatory approval of the experimental cannabidiol (CBD) gel Zygel for fragile X syndrome, according to the company.

Shortage of energy due to leaky mitochondria — the powerhouses of cells — contributes to behavioral abnormalities in fragile X syndrome, a study has found. Closure of the leaky mitochondrial membrane with a form of a Parkinson’s disease medicine boosted energy production and reversed autism-like behaviors…

Measuring the levels of three blood biomarkers could help identify patients with fragile X syndrome who are likely to benefit from treatment with the diabetes medicine metformin, a study suggests. The study, “Blood-Based Biomarkers Predictive of Metformin Target Engagement in Fragile X Syndrome,” was published in the journal…

A parent language intervention based on story-telling eases the severity of speech and social impairments in children with fragile X syndrome, a study shows. The study also found that adding lovastatin, a medication widely prescribed for lowering cholesterol, leads to no additional benefits. The study “Controlled…

Poorer health-related quality of life (HRQoL) correlate with greater emotional, behavioral, and school problems in children with fragile X syndrome, according to a study involving parent questionnaires. The study, “Examination of Correlates to Health-Related Quality of Life in Individuals with Fragile X Syndrome,” was published in the journal…

Genetic testing for fragile X syndrome (FXS) should be reserved as a second-line strategy for people without clinical features or family history suggestive of FXS, according to a Canadian study. Researchers argued that 96% of FXS cases were identified through clinical symptoms and/or family history of FXS. The study…

Zynerba Pharmaceuticals has completed enrollment for the CONNECT-FX trial testing its cannabidiol gel, Zygel, as a treatment for behavioral symptoms in children and teenagers with fragile X syndrome. Recruitment took place at 21 clinical sites in the United States, Australia, and New Zealand. Topline results from…

A Phase 2/3 trial testing the cannabidiol gel Zygel as a treatment for behavioral symptoms in children and teenagers with fragile X syndrome has completed screening for eligible participants. As a result, Zynerba informed clinical sites that screening has closed. The…

Increasing the levels of the SOD2 protein in mitochondria — the powerhouses of cells — may help halt symptoms of fragile X-associated tremor/ataxia syndrome (FXTAS), an early study suggests. The study, “Altered mitochondrial function in cells carrying a premutation or unmethylated full mutation of…

The CONNECT-FX trial testing the cannabidiol (CBD) gel Zygel as a treatment for behavioral symptoms in children and teenagers with fragile X syndrome has nearly completed enrollment. Zynerba, the company developing the gel — which is placed on the skin of the shoulder or upper arm —…