Rett Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: Osteoporosis in RETT Syndrome [NA]
A completed clinical trial conducted in Paris studied osteoporosis in patients with Rett syndrome caused by MECP2 mutations.
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CLINICAL_TRIALS trial_result · 2026-03-31Clinical Trial Update: A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome [Phase 3]
A Phase 3 clinical trial evaluating fenfluramine hydrochloride for Rett syndrome, sponsored by UCB BIOSCIENCES, Inc., is currently not recru…
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FDA approval · 2026-03-27FDA Approved: DAYBUE — Supplemental Application (Acadia Pharmaceuticals Inc.)
FDA is reviewing a supplemental application for Daybue (trofinetide) from Acadia Pharmaceuticals under NDA 217026, related to Rett syndrome …
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome [Phase 3]
A Phase 3 clinical trial is ongoing to evaluate the efficacy of oral NA-921 (Bionetide) versus placebo in females with Rett syndrome; recrui…
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders
Clinical trial update on an intensive multimodal neurorehabilitation program aimed at enhancing neuroplasticity in pediatric neurodevelopmen…
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CLINICAL_TRIALS trial_result · 2026-03-18Clinical Trial Update: Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome [Phase 3]
Phase 3 clinical trial update for TSHA-102 gene therapy in young girls with Rett syndrome, focusing on safety and preliminary efficacy, with…
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EMA other · 2026-03-17EMA Opinion under re-examination: Daybu (trofinetide) — Acadia Pharmaceuticals B.V.
EMA's opinion on Daybu (trofinetide) for Rett syndrome is currently under re-examination following the initial decision.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome [Phase 3]
A Phase 3 clinical trial to evaluate NGN-401, a gene therapy, for Rett syndrome in females is currently recruiting.
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: Repurposing Mirtazapine in Rett Syndrome [Phase 2]
Phase 2 clinical trial evaluating repurposing mirtazapine for Rett syndrome with confirmed MECP2 mutation; currently recruiting.
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Technology-supported Motor Rehabilitation for People With Rett Syndrome [NA]
This clinical trial update describes a study evaluating technology-supported motor rehabilitation for individuals with Rett syndrome; the tr…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: The Rett Syndrome Global Registry
Clinical trial update for the Rett Syndrome Global Registry indicating ongoing recruitment.
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) [NA]
Rush University Medical Center is conducting a web-based intervention (WINGS) for parents of youths with several genetic syndromes, and the …
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CLINICAL_TRIALS research · 2025-12-30Clinical Trial Update: A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) [Phase 3]
TSHA-102 gene therapy is being studied in the REVEAL pivotal phase 3 trial for Rett syndrome in females. The study is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-19Clinical Trial Update: Validating Innovative Biosensors for Rett Autonomic Symptom Tracking
A recruiting study to validate innovative biosensors for tracking autonomic symptoms in Rett Syndrome.
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FDA approval · 2025-12-11FDA Approved: DAYBUE STIX — Original Application (Acadia Pharmaceuticals Inc.)
FDA approves DAYBUE STIX (trofinetide) from Acadia Pharmaceuticals as the original NDA (NDA219884) for Rett syndrome.
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CLINICAL_TRIALS research · 2025-12-02Clinical Trial Update: Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome [Phase 2, Phase 3]
An update on the planned Phase 2/3 trial evaluating NTI164 for Rett syndrome, noting that recruitment has not yet begun.
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CLINICAL_TRIALS research · 2025-10-20Clinical Trial Update: Neurophysiologic Biomarkers in Rett Syndrome
This clinical trial update indicates that a study sponsored by Children’s Hospital of Philadelphia is recruiting to evaluate neurophysiologi…
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CLINICAL_TRIALS trial_result · 2025-10-15Clinical Trial Update: Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) [Phase 1, Phase 2]
This clinical trial update reports on the safety and potential efficacy of TSHA-102 in pediatric females with Rett syndrome in the REVEAL Pe…
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CLINICAL_TRIALS other · 2025-09-03Clinical Trial Update: Obi Medical Robot: Evaluating Effectiveness Related to Usability
A clinical trial update on the Obi Medical Robot evaluating usability-related effectiveness for individuals with upper extremity impairments…
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CLINICAL_TRIALS research · 2025-09-02Clinical Trial Update: Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome [Phase 1]
This update covers a Phase 1 exploratory trial (Rett REVOLUTION) evaluating vorinostat for Rett syndrome, which is currently not recruiting.
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CLINICAL_TRIALS research · 2025-08-20Clinical Trial Update: Gene Editing as a Therapeutic Approach for Rett Syndrome
A clinical trial is recruiting to evaluate gene editing as a therapeutic approach for Rett syndrome.
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FDA approval · 2025-08-05FDA Approved: DAYBUE — Supplemental Application (Acadia Pharmaceuticals Inc.)
FDA approved a supplemental application for Daybue (trofinetide) from Acadia Pharmaceuticals Inc.
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: Parent and Infant Inter(X)Action Intervention (PIXI) [NA]
Clinical trial update for the PIXI Inter(X)Action Intervention (NCT03836300), currently enrolling by invitation across Fragile X Syndrome, A…
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CLINICAL_TRIALS research · 2025-07-16Clinical Trial Update: A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome [Phase 2]
A Phase 2 clinical trial is evaluating an antioxidant cocktail versus placebo in children and adolescents with Rett syndrome, and the study …
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CLINICAL_TRIALS research · 2025-07-01Clinical Trial Update: Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome [EARLY_Phase 1]
A single-dose AAV-MECP2 gene therapy trial in Rett syndrome is in early Phase 1 and currently recruiting to assess safety, tolerability, and…