Discovery fuels hope for Rett syndrome treatment
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, reported last week in the journal Science Translational Medicine , 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. "This work is exciting because it shows another possible way forward when treating Rett syndrome," said Steve Kaminsky, Ph.D., chief science officer of Rettsyndrome.org , which has supported the research. "I am hopeful that this work can be quickly transitioned to compoun...