News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

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…

A ketogenic diet, rich in fats and low in carbohydrates (sugars), was able to reduce the frequency of seizures in a mouse model of fragile X syndrome, a study reports. The diet also decreased daytime activity, with varying effects on body weight depending on the sex and…

People with fragile X syndrome are more likely to develop infections, but are less susceptible to autoimmune disorders than the overall population, a new study found. Taken together, this suggests that the immune system is underactive in this patient population. The study, titled, “The phenotypical implications…

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…

Sleep abnormalities are associated with limitations in vocabulary in very young children with developmental conditions including fragile X syndrome, a study reports. Although preliminary, the results suggest that strategies aimed at improving sleep in young children with developmental disorders might also help improve language skills. The study, “…

Specific cognitive deficits in mothers of children with fragile X syndrome are significantly linked to their genetic profile and are worse in older age, a study has found. The study, “Inhibition deficits are modulated by age…

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 —…

Ovid Therapeutics is expecting to have data from a Phase 2 trial testing OV101 (gaboxadol) in teenagers and young adults with fragile X syndrome later this year. The main goal of the randomized double-blind ROCKET study (NCT03697161) is to assess the safety and tolerability of OV101…

Reducing the level of a protein called ICAM5 in the brain improved memory and eased anxiety in a mouse model of fragile X syndrome, a new study shows. The study, “ICAM5 as a novel target for treating cognitive impairment in Fragile X Syndrome,” was published in the Journal…