DelveInsight’s “Rett Syndrome Market Insights, Epidemiology, and Market Forecast-2032″ 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.

To Know in detail about the Rett Syndrome market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Rett Syndrome Market Forecast

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 (2019-2032)

• 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.

• In March 2023, Melbourne-headquartered Neuren Pharmaceuticals reported that its North American collaborator, Acadia Pharmaceuticals, has obtained approval from the US Food and Drug Administration (FDA) for Neuren’s primary candidate, Daybue (trofinetide), designated for addressing Rett syndrome in both adults and children.

• In February 2023, Unravel Biosciences, Inc., a pharmaceutical firm specializing in developing treatments for intricate diseases through the utilization of a machine-learned model of human health, declared the successful conclusion of its Type B pre-IND meeting with the US Food and Drug Administration (FDA) concerning its RVL001 program targeting Rett Syndrome.

• In February 2023, PharmaTher Holdings Ltd., a prominent provider of specialized ketamine pharmaceuticals, revealed that the United States Food and Drug Administration (FDA) has granted orphan drug designation to KETARX (racemic ketamine) for addressing Rett Syndrome, a rare genetic neurological condition.

• As stated by the International Rett Syndrome Foundation (2023), Rett syndrome affects 1 in every 10,000 female births globally and is even rarer in boys. It is typically identified in children between 6 to 18 months when they start missing developmental milestones or lose previously acquired skills.

• According to the Rett Syndrome Research Trust (2023), approximately 1 in 10,000 girls is born with Rett syndrome. Around 15,000 girls and women in the United States and 350,000 globally are affected by this disorder.

• 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 2019 to 2032. 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 2019–2032 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

Download the report to understand which factors are driving Rett Syndrome epidemiology trends @ Rett Syndrome Epidemiology Forecast

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

Discover more about therapies set to grab major Rett Syndrome market share @ Rett Syndrome Treatment Landscape

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: 2019–2032

• 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 

To know more about Rett Syndrome companies working in the treatment market, visit @ Rett Syndrome Clinical Trials and Therapeutic Assessment

Table of Contents 

1. Rett Syndrome Market Report Introduction

2. Executive Summary for Rett Syndrome

3. SWOT analysis of Rett Syndrome

4. Rett Syndrome Patient Share (%) Overview at a Glance

5. Rett Syndrome Market Overview at a Glance

6. Rett Syndrome Disease Background and Overview

7. Rett Syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Rett Syndrome 

9. Rett Syndrome Current Treatment and Medical Practices

10. Rett Syndrome Unmet Needs

11. Rett Syndrome Emerging Therapies

12. Rett Syndrome Market Outlook

13. Country-Wise Rett Syndrome Market Analysis (2019–2032)

14. Rett Syndrome Market Access and Reimbursement of Therapies

15. Rett Syndrome Market Drivers

16. Rett Syndrome Market Barriers

17.  Rett Syndrome Appendix

18. Rett Syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight 

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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