RETT SYNDROME
Clinical trials for RETT SYNDROME explained in plain language.
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Gene therapy hope for toddlers with rare brain disorder
Disease control Not yet recruitingThis study tests a single dose of a gene therapy called TSHA-102 in 3 girls aged 2 to 4 with Rett syndrome, a severe genetic disorder. The therapy is given as a spinal injection and aims to improve symptoms by delivering a working copy of the faulty gene. The main goal is to chec…
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: Taysha Gene Therapies, Inc. • Aim: Disease control
Last updated May 12, 2026 13:40 UTC
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Cannabis extract shows promise for rare genetic disorder in new trial
Symptom relief Not yet recruitingThis study tests a cannabis plant extract with very low THC (NTI164) in 40 females aged 4-25 with Rett syndrome, a rare genetic condition causing severe physical and mental challenges. Participants will receive either NTI164 or a placebo, then switch after a period. The goal is t…
Matched conditions: RETT SYNDROME
Phase: PHASE2, PHASE3 • Sponsor: Fenix Innovation Group • Aim: Symptom relief
Last updated May 14, 2026 12:03 UTC
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Hope for rett syndrome: new drug trial targets behavior and sleep
Symptom relief Not yet recruitingThis 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 …
Matched conditions: RETT SYNDROME
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Symptom relief
Last updated May 11, 2026 20:46 UTC