AANS Neurosurgeon | Volume 26, Number 3, 2017

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Discovery Fuels Hope for Rett Syndrome Treatment

Vanderbilt University researchers have relieved symptoms of Rett syndrome in a mouse model with a small molecule that works like the dimmer switch in an electrical circuit.

This is the second compound developed by the Vanderbilt Center for Neuroscience Drug Discovery (VCNDD) that relieves Rett syndrome-associated symptoms in mice.

The latest findings provide further evidence that it may be possible one day to develop a drug to treat the rare neurodevelopmental disorder, which occurs predominantly in females.

Further study is needed before this approach can be tested in patients, but animal work from other groups suggests that symptoms in Rett syndrome may be reversible, said senior author Colleen Niswender, Ph.D., associate professor of Pharmacology and VCNDD director of Molecular Pharmacology.

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