Major trial aims to ease debilitating symptoms of rare genetic disorder
Disease control
Not yet recruiting
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 communica…
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Apr 02, 2026 06:57 UTC