Treatment found to improve cognitive function in patients wi
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According to study results published in Nature Medicine, an experimental treatment produced improvements in cognitive function and language in patients with fragile X syndrome. Fragile X syndrome (known as FXS for short) is the most common known genetic cause of autism and the most common cause of inherited intellectual disability.

The study was a phase two clinical trial to assess the safety and efficacy of a drug known as BPN14770 in 30 men between the ages of 18 and 41 years who have fragile X syndrome. BPN1477 inhibits the activity of an enzyme known as phosphodiesterase?4D (PDE4D), which controls the availability in the brain of cyclic adenosine monophosphate (cAMP), a molecule that is critically involved in memory formation. By inhibiting PDE4D, the drug increases the levels of cAMP in the brain.

Participants in the study received daily oral doses of BPN14770 twice a day or a placebo for 12 weeks. Parents, caregivers, and physician raters were kept unaware of whether the participants received the treatment or the placebo. The study evaluated the participants using a version of the National Institutes of Health (NIH) Toolbox.

Cognitive assessments using the NIH Toolbox revealed a significant benefit in oral reading recognition, picture vocabulary and the cognition crystallized composite score. Parent/caregiver ratings revealed a benefit that was judged to be clinically significant in language and daily functioning.

After 12 weeks of treatment in the study, patients crossed over and took a placebo if they had been taking a drug, and a drug if they had been taking a placebo for another 12 weeks. The benefit of BPN14770 was found to persist up to 12 weeks after the crossover from drug to placebo. BPN14770 was very well tolerated, with few adverse events.

This study indicates significant cognitive improvement in domains related to language with the corresponding improvement in caregiver scales rating language and daily functioning.

Nature Medicine