New Version of Bumetanide May Treat Autism While Reducing Side Effects

Bumetanide Has Helped Reduce Autism Symptoms in Children

Italian researchers report that a new small molecule they developed has reduced autism brain pathology in a mouse model of autism. The compound was also effective in reducing the core autism-like social and repetitive behaviors in the valproic acid (VPA) mouse model of ASD. The molecule tested, ARN23746, is a derivative of bumetanide, a common diuretic already approved by the FDA. Bumetanide has been shown in several studies to reduce autism core behaviors in ASD children. However, bumetanide may lead to frequent urination as a side effect. The novel compound does not have this side effect. The study investigators state that ARN23746 is “a solid compound candidate ready for advanced preclinical and manufacturing studies toward development into a clinically relevant drug for unprecedented sustainable therapeutics” for autism as well as other neurological conditions like Down Syndrome.

Original Study

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