Trofinetide for Rett Syndrome in Recent News

Public release: June 22, 2015

A novel agent is holding out some much-needed promise for girls and women with Rett syndrome.

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The new drug, trofinetide, was used in an exploratory phase 2 trial. In this trial Dr. Daniel Glaze explains that, "The new results provide initial evidence that the drug may be an effective treatment for Rett syndrome."

The study was conducted on 56 female patients that were on average about 25 years old. Findings from the study were that both doses of the drug given to the participants were safe and well tolerated after 28 days.

According to Dr. Glaze, "The bottom line is that this study demonstrated that the drug is safe and well tolerated at doses of 35 and 70 mg per kg and it demonstrated that the highest dose exceeded prespecified criteria for clinical benefit," he concluded. "I suggest that these results provide initial evidence that NNZ-2566 may be an effective drug for treating Rett syndrome, and that it supports further studies."

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