Major trial aims to ease debilitating symptoms of rare genetic disorder
NCT ID NCT07503444
Summary
This study is testing whether a medication called fenfluramine hydrochloride can help improve daily life for people with Rett syndrome. Researchers will compare the drug against a placebo in 200 participants aged 5-35 to see if it reduces behavioral challenges, improves communication, and helps with sleep. The study will also carefully monitor for potential heart-related side effects.
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Conditions
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