Zatolmilast, Shionogi’s experimental oral therapy, was not superior to a placebo in improving key measures of cognition, including verbal knowledge and language skills, in boys and men with fragile X syndrome (FXS) in two late-stage clinical trials. The results come from two U.S.-based Phase 3 studies,…
Suppressing the bone morphogenetic protein (BMP) signaling pathway specifically in astrocytes, the star-shaped brain cells that support neurons, may help reduce seizure severity in fragile X syndrome, a mouse study shows. “Seeing that targeting the BMP pathway in astrocytes alleviated some [fragile X] symptoms makes us optimistic about astrocytes…
Fragile X syndrome is a genetic condition associated with cognitive impairment, learning and behavioral challenges, and several physical features. Learn more about the disease here.
Fragile X is caused by mutations in the fragile X mental retardation 1 (FMR1) gene, located in the X chromosome. Learn more about what causes the disease here.
Symptoms differ in severity and occurrence among patients. Life expectancy for fragile X patients is generally normal. Learn more about the symptoms of the disease here.
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