Autism spectrum drugs near clinical testing
The Autism News | English
Researchers at Vanderbilt University are hoping to begin clinical trials soon on compounds they have been developing as possible treatments for neurobehavioral disorders believed to be associated with a genetic abnormality known as fragile X syndrome.
Since 2008 the investigators at the Vanderbilt Center for Neuroscience Drug Discovery have been working with a $4.5 million grant from the private research firm Seaside Therapeutics.
The drug-like molecules the Vanderbilt researchers have been creating are in the final stages of preclinical testing, according to Jeffrey Conn, co-director of the Vanderbilt Center for Neuroscience Drug Discovery. This testing must be successfully completed before the U.S. Food and Drug Administration will approve testing of the molecules on humans.
Conn is a member of the advisory board of Cambridge, Mass.-based Seaside Therapeutics.
Fragile X syndrome, the most common inherited form of intellectual and developmental disabilities, is thought to be the result of gene mutations on the X chromosome, one of the two chromosomes that determine a person’s sex. Women have two X chromosomes and men have one X chromosome and one Y chromosome.
The mutation involved in fragile X syndrome is believed to hinder the production of a protein molecule known as the fragile X mental retardation protein. FMRP is known to play a role in normal cognitive development.
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