News

Using a gene editing tool, researchers successfully reactivated the FMR1 gene — which is silenced in fragile X syndrome patients — in human stem cells. The study, “Targeted reactivation of FMR1 transcription in fragile X syndrome embryonic stem cells” was published in Frontiers in Molecular Neuroscience. Fragile X syndrome (FXS) is…

A small molecule called IGS-1.76 that inhibits excessive interactions between nerve cells could potentially be developed as a therapeutic candidate for fragile X syndrome, a study shows. The study, “Deciphering the Inhibition of the Neuronal Calcium Sensor 1 and the Guanine Exchange Factor Ric8a with a Small Phenothiazine Molecule…

A new pharmacological strategy was able to minimize many cognitive and behavioral defects in mice with fragile X syndrome. Researchers now believe this may be a promising therapeutic strategy for this disease and other brain disorders. The findings from the study, “Isoform-selective phosphoinositide 3-kinase inhibition ameliorates a broad range…

Zynerba Pharmaceuticals’ investigative therapy ZYN002 — a pharmaceutically produced cannabidiol (CBD) gel — was seen to significantly improve behavioral symptoms in children and adolescents with fragile X syndrome (FXS), according to new Phase 2 clinical data. “These data are consistent and compelling, and suggest that ZYN002 may have a clinically…

Ovid Therapeutics has initiated the Phase 2 ROCKET clinical trial to assess the safety, tolerability, and efficacy of OV101 (gaboxadol) as a treatment for adolescents and young adults with fragile X syndrome. The study is expected to enroll about 30 male patients with clinically diagnosed fragile X syndrome, between…

Zynerba Pharmaceuticals has launched a pivotal Phase 3 clinical trial, called CONNECT-FX, that will assess the safety and effectiveness of its cannabis-based ZYN002 as a potential therapy to treat behavioral symptoms in patients with fragile X syndrome. Top-line results from the trial are expected in…

Irritable behavior in patients with fragile X syndrome significantly improved after receiving aripiprazole, an antipsychotic medication previously approved by the U.S. Food and Drug Administration (FDA) to treat other psychotic conditions. The study, “A prospective open-label of aripiprazole in fragile X syndrome,” was published in Psychopharmacology. Fragile…

Invitae Corporation has launched the Invitae Carrier Screen, an affordable test designed to inform prospective parents of genetic changes that increase their risk of having a child with an inherited genetic disorder, such as fragile X syndrome. “By providing affordable, comprehensive, high-quality genetic information, Invitae carrier testing can help parents-to-be…

Chemical modification of a DNA-binding protein may provide a new therapeutic target to treat fragile X syndrome (FXS), a mouse study suggests. The study, “Reducing histone acetylation rescues cognitive deficits in a mouse model of Fragile X syndrome,” was published in …

A gene editing tool based on gold nanoparticles called CRISPR-Gold was able to reduce the repetitive behaviors associated with autism spectrum disorders (ASD) in a mouse model of fragile X syndrome (FXS), a recent study shows. The study, “Nanoparticle delivery of CRISPR into the brain rescues…