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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14th International Conference on Neurology, Neuroscience and Neuromuscular Disorders
June 17-18, 2019; Tokyo
CARS 2019 Computer Assisted Radiology and Surgery
June 18-21, 2019; Rennes, France
18th Meeting of WSSFN
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June 24-28, 2019; Brescia, Italy
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