Lindsey Shapiro, PhD, science writer —

People with fragile X syndrome have significantly lower levels of fatty acids — a type of fatty molecule essential for brain health — in the blood than do healthy people, a study showed. These findings add to data from previous studies reporting lower-than-normal cholesterol levels in this patient population.

A gene therapy using a modified form of an enzyme called diacylglycerol kinase kappa (DGKk) led to the long-term rescue of behavioral symptoms in a mouse model of fragile X syndrome, according to a recent study. Lysogene, which was involved in the studies, is currently developing the…

The University College of London (UCL) has licensed VSN16R, an investigational oral treatment for fragile X syndrome, to U.S.-based Lenire Biosciences. Although originally developed by UCL researchers for treating muscle spasticity in multiple sclerosis, recent preclinical data showed that VSN16R could lessen behavioral symptoms in a…

The European Commission has granted orphan drug designation to Zygel, Zynerba Pharmaceuticals’ cannabidiol (CBD) transdermal gel candidate to treat for behavioral symptoms in people with fragile X syndrome. Orphan designation is assigned to promising therapies for life-threatening, rare diseases affecting fewer than 5 in every 10,000 people in…