RETT SYNDROME
Clinical trials for RETT SYNDROME explained in plain language.
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One-Shot gene therapy trial offers hope for devastating childhood disorder
Disease control Not yet recruitingThis study is testing a single injection of an experimental gene therapy called TSHA-102 in three young girls (aged 2 to under 4 years) with Rett syndrome. The goal is to see if this one-time treatment is safe and if it can help control the symptoms of this serious genetic disord…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated Apr 04, 2026 01:35 UTC
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New drug trial aims to tame rett syndrome symptoms
Disease control Not yet recruitingThis early-stage study is testing a drug called vorinostat to see if it is safe and might help control symptoms of Rett syndrome. Up to 15 girls and young women with Rett syndrome will take the drug at different doses, with each person also getting a placebo for comparison. The m…
Matched conditions: RETT SYNDROME
Phase: PHASE1 • Sponsor: Unravel Biosciences, Inc. • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC
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Major trial aims to ease debilitating symptoms of rare genetic disorder
Disease control Not yet recruitingThis 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…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC
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Hope for rett syndrome: cannabis oil trial aims to ease devastating symptoms
Disease control Not yet recruitingThis study is testing whether a special cannabis plant extract oil, called NTI164, can help control symptoms of Rett syndrome. Rett syndrome is a rare and severe genetic disorder that mostly affects girls, causing problems with movement, communication, and learning. The trial wil…
Matched conditions: RETT SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Fenix Innovation Group • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC