New research suggests that medication could bring about significant improvements in cognitive function and language in adults with fragile X syndrome, the most common cause of inherited intellectual disability.

A drug known as BPN14770 led to benefits in oral reading recognition, picture vocabulary and overall cognition in adults with fragile X, according to findings published recently in the journal Nature Medicine. In addition, parents and caregivers reported marked improvements in language and daily functioning when adults with fragile X took the medication.

“These results offer hope for patients with fragile X syndrome and their families,” said Elizabeth Berry-Kravis, a pediatric neurologist at Rush University Medical Center and principal investigator of the study. “The majority of clinical outcome measures were in favor of the drug. These measures included performance-based assessments, biomarkers and parent and physician-rated scales, which in combination, suggest a meaningful impact on the global FXS disease process.”

The phase two clinical trial included 30 men ages 18 to 41 with fragile X syndrome. The men were given BPN14770 or a placebo twice each day for 12 weeks and then they swapped to whichever treatment they didn’t receive initially for the next 12 weeks.

The men were assessed using a version of the National Institutes of Health Toolbox Cognitive Battery that was designed for those with intellectual disability. In addition, the participants’ parents reported on their status.

“This is the first time that the NIH Toolbox has been able to be used to demonstrate a cognitive change in a trial in people with intellectual disabilities,” Berry-Kravis said. “In just three months, we saw improvement specifically in the verbal subtests of the NIH Toolbox, coupled with parent rating of improvements, particularly in language.”

Researchers said that BPN14770 was well tolerated and remained effective for up to 12 weeks after individuals stopped taking it. They said further study is needed in a larger group to fully assess the cognitive benefit of the drug.

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