STAT+: FDA approves first treatment for Rett syndrome, a genetic neurological disease
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.
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