RETT SYNDROME
Clinical trials for RETT SYNDROME explained in plain language.
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New hope for rett syndrome? early drug trial begins
Disease control Not yet recruitingThis early-stage study is testing a drug called vorinostat to see if it is safe and can help control symptoms of Rett syndrome. Up to 15 girls and young women with Rett syndrome will take the drug for several months, with each person's results compared to their own baseline. The …
Matched conditions: RETT SYNDROME
Phase: PHASE1 • Sponsor: Unravel Biosciences, Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:42 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
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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 with Rett syndrome. The goal is to see if the treatment is safe and if it can help control the disease by targeting its genetic cause. Participants will be followed close…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated Mar 23, 2026 15:16 UTC