080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Antagonizing fragile X

Emory University |

Nearly three decades ago, an Emory-led international team identified fragile X syndrome as the most common inherited cause of intellectual disability.

Emory human genetics chair Stephen Warren and his colleagues discovered the mutated gene on the X chromosome that causes this disorder. The scientists helped develop a screening test for fragile X and have been studying it ever since.

But until now there has been no treatment. That may soon change. Along with four other medical centers, Emory is participating in a phase 2 clinical trial that will test a targeted drug therapy for fragile X syndrome. The randomized, double-blind study has 32 adult participants from 18 to 50 years, who have fragile X syndrome. A second study will continue testing the most effective dose of the drug against a placebo in 28 participants.


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