Hope for rett syndrome: new drug trial targets behavior and sleep
Symptom relief
Not yet recruiting
This study tests whether fenfluramine hydrochloride can improve symptoms of Rett syndrome, a rare genetic disorder that affects mostly girls. About 200 participants will receive either the drug or a placebo for 14 weeks, with an option to continue on the drug afterward. The main …
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Symptom relief
Last updated May 11, 2026 20:46 UTC