FRAXA Research Foundation is supporting a Phase 2b clinical trial of SPG601, an experimental therapy targeting BK channels in fragile X syndrome, following promising early results.
Treating Fragile X might require a combination of drugs. FRAXA-DVI tested ibudilast and gaboxadol in Fragile X mice. Together they rescued a wide array of symptoms.
Girls and women with Fragile X syndrome show different learning impairments relative to boys and men with the disease, a finding that was paralleled in a mouse model of the disease, a study found.
Behavioral problems such as hyperactivity and aggression are more frequent in children with Fragile X syndrome, becoming less common as they grow through adolescence into adulthood.
Zynerba Pharmaceuticals received advice from the U.S. Food and Drug Administration (FDA) on the design of a Phase 3 clinical trial of Zygel as a cannabidiol treatment for Fragile X syndrome.
A study funded by FRAXA in Italy has encouraging results for people with Fragile X: drugs that block adenosine receptors (A2A) reversed signs of Fragile X in a mouse model.
Zynerba presented clinical trial results for Zygel at a recent neurology conference. Zygel, an experimental cannabidiol (CBD) gel, may reduce behavioral abnormalities in children with Fragile X syndrome who have more severe disease.
Tetra Therapeutics and Shionogi announced plans to expand their partnership supporting BPN14770, a treatment candidate for disorders marked by cognitive and memory deficits.
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 of immune dysregulation in Fragile X syndrome,” was published in the European Journal of Neurology.
First Healx secured $56M in new financing to launch a global Rare Treatment Accelerator program to tackle Fragile X syndrome and 39 other rare diseases. Now they have built a partnership with Boehringer Ingelheim worth millions. It all started with a small FRAXA grant to Healx to repurpose available drugs for Fragile X.
Dr. Craig Erickson and colleagues at the University of Cincinnati used eye-tracking technology to understand sociability in Fragile X syndrome. This study affirms what so many parents, caretakers, and educators suspect: people with fragile X want to be social, and it is anxiety – not lack of interest – which usually hold them back. If anxiety could be reduced, more sociability would likely follow. Dr. Erickson is a Fragile X expert and FRAXA investigator who is currently conducting a Fragile X clinical trial of an investigational new drug.
Patients with Fragile X syndrome who don’t meet the cut-off for a diagnosis of autism show a decrease in impulsivity and repetitive questioning over time, when compared with patients who do, a new study shows.
