Rett Syndrome
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CLINICAL_TRIALS research · 2025-06-12Clinical Trial Update: Cognitive Function in Rett Syndrome During Trofinetide Treatment
A clinical trial update for Rett syndrome evaluating cognitive function during treatment with trofinetide; the study is currently recruiting…
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CLINICAL_TRIALS trial_result · 2025-03-11Clinical Trial Update: GCB-002 in Treatment of Patients With Rett Syndrome [NA]
This document reports an ongoing clinical trial evaluating GCB-002 for Rett syndrome with MECP2 mutation. Enrollment is by invitation.
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CLINICAL_TRIALS trial_result · 2025-02-27Clinical Trial Update: To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome [Phase 2, Phase 3]
This clinical trial update reports the completion of phase 2 and phase 3 studies evaluating the efficacy and safety of oral NA-921 (Bionetid…
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CLINICAL_TRIALS research · 2025-02-18Clinical Trial Update: Esketamine for the Treatment of Rett Syndrome [EARLY_Phase 1]
An early-phase (Phase 1) trial is evaluating esketamine for Rett syndrome, sponsored by the Children's Hospital of Fudan University; the stu…
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CLINICAL_TRIALS trial_result · 2024-11-14Clinical Trial Update: ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
A ClinicalTrials.gov update indicating the study assessing Emerald and MC10 BioStamp nPoint biosensors for Rett syndrome has completed.
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CLINICAL_TRIALS research · 2024-10-03Clinical Trial Update: Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome [NA]
A Georgetown University-led clinical trial is evaluating whether computer gaming can improve arm use in Rett syndrome and is currently recru…
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CLINICAL_TRIALS research · 2024-10-01Clinical Trial Update: Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT) [Phase 1, Phase 2]
Update on a Phase 1/2 clinical trial evaluating NTI164, a full-spectrum cannabis plant extract with 0.08% THC, for Rett Syndrome; the study …
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FDA approval · 2024-09-30FDA Approved: DAYBUE — Supplemental Application (Acadia Pharmaceuticals Inc.)
The FDA approved a supplemental application for DayBUE (trofinetide) from Acadia Pharmaceuticals Inc.
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CLINICAL_TRIALS trial_result · 2024-09-27Clinical Trial Update: Open-Label Extension Study of Trofinetide for Rett Syndrome [Phase 3]
Open-label extension study of trofinetide for Rett syndrome was terminated (Phase 3), with results posted on 2024-09-27.
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CLINICAL_TRIALS trial_result · 2024-09-24Clinical Trial Update: An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome [Phase 2, Phase 3]
An open-label Phase 2/3 trial of trofinetide for Rett syndrome in girls aged 2 to 5 was terminated, with results posted in 2024.
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CLINICAL_TRIALS trial_result · 2024-04-11Clinical Trial Update: Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome [Phase 3]
Open-label extension study results for trofinetide in Rett syndrome completed with results posted in 2024.
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CLINICAL_TRIALS trial_result · 2024-04-08Clinical Trial Update: Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™) [Phase 3]
Phase 3 clinical trial of trofinetide for Rett syndrome has completed; results posted in 2024.
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CLINICAL_TRIALS trial_result · 2024-03-27Clinical Trial Update: A Study to Evaluate Ketamine for the Treatment of Rett Syndrome [Phase 2]
A Phase 2 clinical trial evaluating ketamine for Rett syndrome has completed, with results posted.
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CLINICAL_TRIALS trial_result · 2023-08-21Clinical Trial Update: ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome [Phase 2, Phase 3]
Clinical trial update for ANAVEX2-73 in pediatric Rett syndrome has completed Phase 2/3 studies.
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FDA approval · 2023-03-10FDA Approved: DAYBUE — Original Application (Acadia Pharmaceuticals Inc.)
FDA approved DAYBUE (trofinetide) from Acadia Pharmaceuticals as the first approved treatment for Rett syndrome.
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CLINICAL_TRIALS trial_result · 2022-11-23Clinical Trial Update: Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients [Phase 2]
Phase 2 clinical trial update for VYNT-0126 in adults with Rett Syndrome.
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CLINICAL_TRIALS trial_result · 2022-08-08Clinical Trial Update: A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome [Phase 3]
Phase 3 long-term safety study of cannabidiol oral solution in Rett syndrome was terminated; results posted in 2022.
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CLINICAL_TRIALS trial_result · 2022-01-27Clinical Trial Update: ANAVEX2-73 Study in Patients With Rett Syndrome [Phase 3]
Phase 3 clinical trial update for ANAVEX2-73 in Rett syndrome has completed.
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CLINICAL_TRIALS trial_result · 2021-12-29Clinical Trial Update: Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome [Phase 3]
Phase 3 trial of cannabidiol oral solution (GWP42003-P, CBD-OS) in Rett syndrome has been terminated with results posted.
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CLINICAL_TRIALS trial_result · 2021-12-21Clinical Trial Update: Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms [Phase 2, Phase 3]
Clinical trial update for sarizotan in Rett syndrome with respiratory symptoms has terminated; results posted previously.
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CLINICAL_TRIALS trial_result · 2021-01-12Clinical Trial Update: Study of ANAVEX2-73 in Patients With Rett Syndrome [Phase 2]
Phase 2 clinical trial evaluating ANAVEX2-73 in Rett syndrome has completed.
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CLINICAL_TRIALS trial_result · 2020-08-14Clinical Trial Update: A Safety Study of NNZ-2566 in Pediatric Rett Syndrome [Phase 2]
Phase 2 safety study of NNZ-2566 in pediatric Rett syndrome has completed.
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CLINICAL_TRIALS trial_result · 2020-07-17Clinical Trial Update: Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin [Phase 2]
A Phase 2 clinical trial evaluating triheptanoin for Rett syndrome is ongoing, with status currently unknown.
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CLINICAL_TRIALS trial_result · 2019-05-07Clinical Trial Update: Pharmacological Treatment of Rett Syndrome With Statins [Phase 2]
Phase 2 clinical trial evaluating statins for pharmacological treatment of Rett syndrome has completed with results posted.
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CLINICAL_TRIALS trial_result · 2019-02-21Clinical Trial Update: 'Uptime' Participation Intervention in Girls and Women With Rett Syndrome [NA]
The Rett syndrome study 'Uptime' Participation Intervention conducted by Rigshospitalet (Denmark) has completed. No specific results are pro…