Rett syndrome Advocacy Hub | UniteRare

At a Glance

Live from database

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Regulatory Watchboard

10 events

Jul 2026Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

Fenix Innovation Group — PHASE2, PHASE3

TrialNOT YET RECRUITING

Mar 2026A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

UCB BIOSCIENCES, Inc. — PHASE3

TrialNOT YET RECRUITING

Mar 2026Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome

Taysha Gene Therapies, Inc. — PHASE3

TrialNOT YET RECRUITING

Oct 2025To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Biomed Industries, Inc. — PHASE3

TrialRECRUITING

Oct 2025Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome

Unravel Biosciences, Inc. — PHASE1

TrialNOT YET RECRUITING

Oct 2025A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome

Holland Bloorview Kids Rehabilitation Hospital — PHASE2

TrialNOT YET RECRUITING

Jul 2025Repurposing Mirtazapine in Rett Syndrome

University of Trieste — PHASE2

TrialRECRUITING

Mar 2025Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

Rett Syndrome Research Trust

TrialRECRUITING

Jan 2025Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

Guangzhou Women and Children's Medical Center — EARLY_PHASE1

TrialRECRUITING

Dec 2024Cognitive Function in Rett Syndrome During Trofinetide Treatment

University of Minnesota

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

Total programs

Open now

Patient Assistance Programs1

Daybue

Acadia Pharmaceuticals Inc.

OpenContact for details

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Approved Treatments

1 FDA-approved

Daybue

(TROFINETIDE)Orphan drugstandard

Acadia Pharmaceuticals Inc.

12.1 Mechanism of Action The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown.

FDA label ↗

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Clinical Trial Landscape

17 active trials

3Phase 3

1Phase 2

3N/A

6Unknown

2PHASE1, PHASE2

2EARLY_PHASE1

17Total recruiting

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Recent News & Research

No recent news articles indexed yet for Rett syndrome.
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Specialist Network

Top 6 by expertise

Alan Percy, MD, M.D

University of Alabama at Birmingham

📍 BIRMINGHAM, AL

Score 94 trials

Jeffrey Neul, MD, PhD, M.D., Ph.D

Vanderbilt University Medical Cener

📍 NASHVILLE, TN

Score 32 trials

Mustafa Sahin, MD, PhD, MD

Boston Children's Hospital

📍 BOSTON, MA

Score 35 trials

Barbara B Biesecker, CRNP

National Human Genome Research Institute (NHGRI)

📍 CLARKS SUMMIT, PA

Score 316 trials

Jeffrey L Neul, MD, PhD, M.D., Ph.D

Vanderbilt University

📍 NASHVILLE, TN

Score 22 trials

Rosa Angela Fabio

Department of Clinical and Experimental Medicine, University of Messina

Score 01 trial

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