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The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement.

The new drug, called Daybue, is made by Acadia Pharmaceuticals. It’s not a cure, but in a clinical trial, the drug improved behavioral symptoms associated with Rett. Daybue also causes high rates of diarrhea and vomiting, which may impact its use.


The approval of Daybue is an “historic moment,” said Melissa Kennedy, CEO of the International Rett Syndrome Foundation, a patient advocacy group. “We thank the courageous families who participated in the clinical trials. It’s because of them and their bravery that the Rett syndrome community celebrates this milestone. And we’re grateful for the investments made by Acadia.”

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