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12:00 AM
 | 
Nov 02, 2009
 |  BioCentury  |  Product Development

Stabilizing fragile X

It has long been thought that excessive protein synthesis plays a role in neurological conditions like fragile X syndrome, though little progress has been made in identifying therapeutic targets. Seaside Therapeutics LLC thinks it has found one in metabotropic glutamate receptor 5 and on Monday was to announce the start of a Phase I trial of an in-licensed small molecule antagonist.

Fragile X syndrome is a heritable form of mental retardation and one of the foremost known causes of autism, according to Randall Carpenter, president and CEO of Seaside. The condition is characterized by impaired neural and cognitive development caused by transcriptional silencing of the FMR1 gene, which encodes the fragile X mental retardation protein (FMRP).

While aberrant protein synthesis has been suggested as a cause of a variety of neurological disorders, finding druggable targets has been easier said...

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