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Trofinetide’s impact on Rett syndrome (Key points)
Improves core symptoms of Rett syndrome
Trofinetide has shown modest but statistically significant improvement in core symptoms of Rett syndrome, especially in communication, behavior, and daily functioning compared to placebo in clinical trials. (Nature)
Works on overall clinical improvement
Patients receiving trofinetide showed better global clinical scores, including caregiver and physician-rated improvement scales, indicating overall functional benefit. (PubMed)
Generally safe but causes common side effects
The drug is considered safe and well-tolerated, but the most frequent side effects are diarrhea, vomiting, and reduced appetite, which can affect quality of life. (Nature)
If you want, I can also explain it in very simple language or exam notes format 👍
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