Brain Defect Helps Drive Fragile ... (en have a permanent defect in their DNA ...)

Brain Defect Helps Drive Fragile X Syndrome

Date:9/20/2007

en have a permanent defect in their DNA," he said. "The goal here is to improve the quality of life for these children. We are going to decrease the severity of episodes to help them focus better on learning tasks and help with the behavior problems and improve their cognitive function," he said.

One expert hailed the finding.

"This is a very important paper," said Dr. Randi Hagerman, a professor of pediatrics and medical director of the M.I.N.D. Institute at the University of California, Davis. "It proves that mGluR5 antagonists will be helpful in kids with fragile X syndrome," she added. "We are looking forward to a new age of treatment in fragile X syndrome."

Hagerman noted that trials with mGluR5 antagonists on adults with fragile X syndrome will be starting this fall. "If things go well with adults, then we will move to pediatric trials," she said.

"This is a very hopeful message," Hagerman said. "This means that there will be very specific treatments that will have an impact in the very near future."

Another expert agreed that the discovery should lead to new treatments for children with fragile X.

"This is a very exciting paper, which is a powerful confirmation of the mGluR theory of fragile X," said Dr. Michael Tranfaglia, medical director of the FRAXA Research Foundation. "Since FRAXA Research Foundation is currently working with several pharmaceutical companies to bring mGluR5 antagonists into clinical trials for fragile X, we are delighted to see this elegant proof of the therapeutic potential of this class of drugs."

More information

For more information on fragile X syndrome, visit the Fragile X Research Foundation.

SOURCES: Gary J. Bassell, Ph.D., professor, cell biology, Emory University, Atlanta; Randi Hagerman, M.D., professor, pediatrics, and medical director, M.I.N.D. Institu
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