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Drug candidate fails to improve symptoms of fragile X syndrome

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2:00pm, January 13, 2016
Fragile X mutation

A mutation on the X chromosome, above, causes fragile X syndrome, which can cause intellectual disabilities and autism spectrum disorders. An experimental drug for treating the syndrome has proven ineffective in clinical trials.  

An experimental drug for the genetic disorder fragile X syndrome was ineffective in two studies in people.

The syndrome, which is caused by a mutation on the X chromosome, can cause intellectual disability, attention deficit disorder and autism spectrum disorders.

The drug candidate, called mavoglurant, had shown promise in mice with a genetic condition similar to fragile X. The drug suppresses the activity of the protein mGluR5, thought to play a role in altering brain cell structure in patients with fragile X syndrome (SN: 6/2/12, p. 17).

But in 12-week clinical trials in people, mavoglurant did not improve symptoms such as irritability or hyperactivity in 175 adults and 139 adolescents with fragile X syndrome, researchers report in the Jan. 13 Science Translational Medicine

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