Rett Syndrome Market Trends Point to Steady Growth Ahead by 2034, DelveInsight Analysis Reveals

DelveInsight's "Rett Syndrome Market Insights, Epidemiology, and Market Forecast-2034′′ report offers an in-depth understanding of the Rett Syndrome, historical and forecasted epidemiology as well as the Rett Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Rett Syndrome Market Report:
• The Rett Syndrome market size is anticipated to grow with a significant CAGR during the study period (2020-2034).
• In September 2025, Interim results from the ongoing Phase IV LOTUS trial revealed that Acadia Pharmaceuticals' Daybue (trofinetide) delivered significant behavioral improvements and enhanced quality of life in patients with Rett syndrome. The company noted that these outcomes strengthen confidence in Daybue as the first FDA-approved therapy for Rett syndrome and underscore its potential to address long-standing unmet needs for both patients and caregivers.
• In June 2025, Neurogene Inc. (Nasdaq: NGNE), a clinical-stage biotech established to develop transformative genetic therapies for patients and families impacted by rare neurological disorders, announced details of EmboldenTM, its registrational clinical trial evaluating NGN-401 gene therapy in females with Rett syndrome aged 3 years and older. The company has secured written agreement from the U.S. Food and Drug Administration (FDA) on critical elements of the trial's design.
• In May 2025, Neurogene has implemented a new monitoring and treatment protocol for its adeno-associated virus (AAV) gene therapy following the death of a Rett syndrome patient in a clinical trial. Announced at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, the company stated that the algorithm is designed to counteract hemophagocytic lymphohistiocytosis (HLH)-a rare but severe hyperinflammatory syndrome linked to high systemic exposure from elevated doses of AAV gene therapy.
• In May 2025, Taysha secured FDA agreement on the pivotal Part B trial design for TSHA-102, allowing the company to submit an Investigational New Drug (IND) amendment without the need for a formal end-of-phase meeting. An update on the program-including details on the pivotal design, safety and efficacy results, and a Rett syndrome natural history analysis-is expected in the second quarter of 2025 at the International Rett Syndrome Foundation (IRSF) Meeting. The company also highlighted that TSHA-102 could be eligible for a Priority Review Voucher, which may accelerate its regulatory review process.
• In January 2025, Acadia Pharmaceuticals submitted its first Marketing Authorization Application (MAA) in Europe to the European Medicines Agency (EMA) for trofinetide, aimed at treating Rett syndrome in patients aged two years and above. If granted approval, trofinetide would become the first approved therapy for Rett syndrome in the EU.
• In November 2024, Neurogene Inc. (Nasdaq: NGNE), a clinical-stage biotech focused on creating genetic therapies for rare neurological conditions, announced encouraging interim findings from its ongoing Phase 1/2 open-label study. The trial, assessing NGN-401 gene therapy in young female patients with Rett syndrome, revealed promising outcomes in the initial four participants of the low-dose group, highlighting a favorable safety profile.
• In October 2024, Unravel Biosciences, Inc. ("Unravel"), an AI-powered therapeutics company dedicated to advancing treatments for complex diseases, has teamed up with the clinical research division of PECET at the University of Antioquia in Medellín, Colombia. Accredited in Good Clinical Practices by the Colombian Health Regulatory Authority (INVIMA), PECET will serve as the clinical site for Unravel's RVL-001 proof-of-concept trials, which aim to address Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS).
• In November 2024, Neurogene Inc. (Nasdaq: NGNE), a clinical-stage biotechnology company dedicated to developing genetic medicines for rare neurological diseases, has announced positive interim results from the first four participants in the low-dose cohort of its ongoing Phase I/II open-label trial evaluating NGN-401 gene therapy for female pediatric patients with Rett syndrome. The low-dose NGN-401 has shown a favorable safety profile in early findings.
• In October 2024, Unravel Biosciences, Inc. ("Unravel"), an AI-driven therapeutics company focused on advancing treatments for complex diseases, has finalized a clinical trial agreement with the clinical research unit of PECET at the University of Antioquia. Certified in Good Clinical Practices by the Colombian Health Regulatory Authority (INVIMA) and based in Medellín, Colombia, PECET will serve as the designated study site for Unravel's RVL-001 proof-of-concept clinical trials targeting Rett syndrome (RTT) and Pitt Hopkins syndrome (PTHS).
• In May 2024, Neurogene released preliminary safety and tolerability findings from its ongoing Phase I/II clinical trial of NGN-401, a gene therapy candidate for Rett syndrome. This open-label study aims to evaluate the safety, tolerability, and early efficacy of NGN-401, which is administered as a one-time intracerebroventricular (ICV) infusion across two dosage cohorts.
• In January 2024, Anavex Life Sciences Corp. announced topline results from the Phase II/III EXCELLENCE clinical trial, a randomized, double-blind, placebo-controlled study that assessed the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) aged 5 to 17 years.
• Rett syndrome affects approximately 1 in every 10,000 female births globally and is extremely rare in boys. It is typically identified in children between 6 to 18 months when they start missing developmental milestones or lose previously acquired skills.
• The prevalence of Rett syndrome in Japan is around 0.90 per 10,000 girls.
• The majority of Rett syndrome patients were in the 18-29 age group, accounting for 25% of the population, followed by the 11-17 group at 21%, the 5-10 group at 20%, with the remaining cases spread across other age groups.
• Key Rett Syndrome Companies: Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others
• Key Rett Syndrome Therapies: Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others
• The Rett Syndrome epidemiology based on gender analzyed that, Rett syndrome occurs more in girls than boys
• The Rett Syndrome market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Rett Syndrome pipeline products will significantly revolutionize the Rett Syndrome market dynamics.

Rett Syndrome Overview
Rett Syndrome is a rare genetic neurological disorder that primarily affects girls. It is caused by mutations in the MECP2 gene on the X chromosome. The syndrome typically manifests after 6-18 months of seemingly normal development, followed by a period of rapid regression in motor, cognitive, and social skills.

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Rett Syndrome Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Rett Syndrome Epidemiology Segmentation:
The Rett Syndrome market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of Rett Syndrome
• Prevalent Cases of Rett Syndrome by severity
• Gender-specific Prevalence of Rett Syndrome
• Diagnosed Cases of Episodic and Chronic Rett Syndrome

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Rett Syndrome Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Rett Syndrome market or expected to get launched during the study period. The analysis covers Rett Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Rett Syndrome Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Rett Syndrome Therapies and Key Companies
• Pridopidine: Prilenia Therapeutics
• GXV-001: GEXVal
• NGN-401: Neurogene Inc
• TSHA-102: Taysha Gene Therapies
• VYNT-0126: Vyant Bio, Inc
• Trofinetide: Neuren Pharmaceuticals
• ANAVEX2-73 (blarcamesine): Anavex Life Sciences
• Vatiquinone: PTC Therapeutics
• Fingolimod: Novartis
• Triheptanoin: Ultragenyx Pharmaceutical
• Cannabidivarin: GW Pharmaceuticals
• STRX 230: StrideBio
• AMO 04: AMO Pharma
• Pridopidine: Prilenia Therapeutics
• NLX 101: Neurolixis
• BHV 5000: Biohaven Pharmaceuticals

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Rett Syndrome Market Strengths
• The use of off-label branded and generic prescription medications targeted at individual symptoms of Rett syndrome, including antiepileptics, antipsychotics, antidepressants and benzodiazepines.
• Several academic institutions and pharmaceutical companies are currently conducting clinical trials for the treatment of various symptoms of Rett syndrome.

Rett Syndrome Market Opportunities
• New therapeutic options are needed to address the unmet need for effective and curative therapies for Rett syndrome. This has created an opportunity for key players to develop novel therapies targeted towards this indication.

Scope of the Rett Syndrome Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Rett Syndrome Companies: Prilenia Therapeutics, GEXVal, Neurogene Inc, Taysha Gene Therapies, Vyant Bio, Inc, Neuren Pharmaceuticals, Anavex Life Sciences, PTC Therapeutics, Novartis, Ultragenyx Pharmaceutical, GW Pharmaceuticals, StrideBio, AMO Pharma, Prilenia Therapeutics, Neurolixis, Biohaven Pharmaceuticals, and others
• Key Rett Syndrome Therapies: Pridopidine, GXV-001, NGN-401, TSHA-102, VYNT-0126, Trofinetide, ANAVEX2-73 (blarcamesine), Vatiquinone, Fingolimod, Triheptanoin, Cannabidivarin, STRX 230, AMO 04, Pridopidine, NLX 101, BHV 5000, and others
• Rett Syndrome Therapeutic Assessment: Rett Syndrome current marketed and Rett Syndrome emerging therapies
• Rett Syndrome Market Dynamics: Rett Syndrome market drivers and Rett Syndrome market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Rett Syndrome Unmet Needs, KOL's views, Analyst's views, Rett Syndrome Market Access and Reimbursement

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