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Rett Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Rett Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Rett Syndrome trials you may qualify forThis observational research study is designed to learn more about how the brain processes sound in individuals with Rett syndrome. Participants will complete a…
The RETT REVOLUTION trial is a placebo-controlled, single-blinded, exploratory study with patients serving as their own control ("N of 1" trial design) where th…
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective…
This observational study evaluates functional and developmental outcomes in pediatric participants undergoing a two week intensive multimodal neurorehabilitatio…
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) i…
Based on our clinical observations, many girls with RETT syndrome, a severe neuro-developmental encephalopathy, suffer from osteoporosis which can appear at a v…
The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).