Atypical Rett syndrome
MONDO:0017746A neurodevelopmental disorder that is diagnosed when a child presents with a Rett-like syndrome but does not fulfill all the diagnostic criteria for typical Rett syndrome (classic/typical RTT).
Also known as: Rett syndrome variant, atypical RTT, Rett like syndrome
12 clinical trials for this condition and its sub-types, 0 tagged with Atypical Rett syndrome itself.
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Browse by category →Sub-types of Atypical Rett syndrome
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FOXG1 disorder 2 trials
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Gene therapy breakthrough? new trial targets rare brain disorder FOXG1
Disease control By invitation onlyThis early-stage trial tests a new gene therapy called FRF-001 for FOXG1 syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the brain's fluid spaces. The study will include 12 children and adults and will check for sa…
Phase 1/2 • Sponsor: FOXG1 Research Foundation • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
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New hope for rare epilepsy: drug shows promise in phase 3 trial
Disease control OngoingThis Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…
Phase 3 • Sponsor: Zogenix, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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New program aims to ease burden on families of kids with rare diseases
Symptom relief By invitation onlyThis study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …
Sponsor: Children's National Research Institute • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC
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New registry aims to unlock secrets of rare CDKL5 disorder
Knowledge-focused PausedThis study creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 UTC