Rett Syndrome
📧 Sign up to get email alerts when something new happens for Rett Syndrome.
Sign up freeTop drugs studied for this disease
-
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.
-
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…
-
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…
-
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…
-
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.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
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…
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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…
-
CLINICAL_TRIALS trial_result · 2018-11-05Clinical Trial Update: Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) [Phase 2]
Phase 2 trial evaluating glatiramer acetate (Copaxone) for Rett syndrome has completed with results posted.
-
CLINICAL_TRIALS trial_result · 2018-10-23Clinical Trial Update: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome [Phase 2]
Phase 2 placebo-controlled trial of dextromethorphan in Rett syndrome completed; results posted in 2018.