This is a summary of an article about the LAVENDER study, which was published in Nature Medicine in June 2023. The study involved girls and young women with a rare genetic condition called Rett syndrome, which affects the way the brain develops. Researchers wanted to find out if a drug called trofinetide could improve the symptoms of Rett syndrome. This Plain Language Summary of Publication article from Future Rare Diseases discusses a rare genetic condition, that affects girls and young women, called Rett syndrome. The syndrome affects the way the brain develops. The LAVENDER study investigated how the medication, trofinetide, could improve several important symptoms of the syndrome; it has been the first study to show that a medicine improves the symptoms of the Rett syndrome.

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The original article on which this summary is based is called ‘Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study’ and was published in Nature Medicine. 

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