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Symptoms Of Fragile X May Be Reversible

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A new study indicates that medication can reverse many of the defining symptoms of fragile X syndrome in mice, a finding which could have implications for people with other developmental disabilities too.

The development reported Thursday in the journal Neuron is significant, scientists say, because the treatment brought about major changes even in adult mice with fragile X, the most common form of inherited intellectual disability.

For the study, researchers looked at a new drug intended to target a protein in the brain that’s been linked to fragile X. Mice given the medication began exhibiting fewer symptoms of the condition, researchers said. Improvements were seen in learning and memory capabilities as well as behavioral areas, with the mice becoming less sensitive to sound, for example.

“The most important implications of our study are that many aspects of (fragile X) are not caused by an irreversible disruption of brain development,” said Mark Bear of the Massachusetts Institute of Technology who worked on the study.

Though there is no plan currently to try the drug used in the study with people, researchers say that the ability to see such a reversal of symptoms in mice is promising. What’s more, they say that other medications already in human clinical trials address the same protein and may be able to offer similar results.

The findings could have significance beyond fragile X as well. Many with the condition also have autism and intellectual disability, so treatments for fragile X may offer a window into the other disorders.

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Comments (3 Responses)

  1. Hannah Jacobs says:

    Why do they have no plans to use the drug in the study in people? Why aren’t they naming the drug?

  2. Tania Morse says:

    Hannah, the drug is likely still in the early experimental stages. An experimental drug must be tested for a certain amount of time and the results of those studies must prove a certain degree of safety before it can legally be tested on people. While the drug may ultimately be tested on people in the future, testing it on people now could have severe consequences. Also, in the experimental stages, drugs often go by a code number of some sort. It would be premature to name a drug that may or may not make it to market.

  3. Cori Kessler says:

    I think this is a great medical break through. I hope the drug will a success. this could be a great benifit to my son some day and to us all. Keep it up.

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