An experimental drug being developed by Acadia Pharmaceuticals improved the symptoms of women and girls with Rett syndrome, a rare genetic disease that robs patients of the ability to interact socially and can cause seizures and other neurological problems, the company said Monday.
The data were made public in a press release and have not yet been peer-reviewed.
Acadia said that the treatment, trofinetide, improved symptoms in the study of 187 girls and young women based on two different surveys, one filled out by health care providers and the other by patients’ families. Rett occurs mainly in girls, with a frequency of one in every 10,000 to 15,000 births. About 6,000 to 9,000 patients have the disease.
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