DelveInsight’s “Rett Syndrome Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Rett Syndrome market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Rett Syndrome drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Rett Syndrome treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Rett Syndrome: An Overview
Rett syndrome is a unique neurodevelopmental disorder that is first noticed in infancy and primarily affects girls, but can be rarely seen in boys. It is caused by mutations in the X-chromosome on a gene called MECP2. There are more than 200 different mutations found on the MECP2 gene. Most of these mutations are found in eight different “hot spots.” Rett syndrome is mostly misdiagnosed as autism, cerebral palsy, or global developmental delay. It is found in all ethnic and racial groups.
Rett syndrome is not a degenerative disorder but causes problems in brain function that are responsible for cognitive, sensory, emotional, motor, and autonomic functions. These can include learning, speech, sensory sensations, mood, movement, breathing, cardiac function, and even chewing, swallowing, and digestion. Symptoms of Rett syndrome appear after an early period of apparently normal or near normal development until six to eighteen months of life when there is a slowing down or stagnation of skills.
Rett Syndrome Market Key Facts
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As per the study titled “Development of consensus-based guidelines for managing communication of individuals with Rett syndrome” by Townend et al., stated that Rett syndrome is a neurological disorder known to occur in approximately 1 in 10,000 females and more rarely in males.
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As per a study conducted by M. Itoh et al., which is titled “Epidemiological study of Japanese Rett syndrome”, the prevalence of Rett Syndrome in Japan is approximately 0.90 per 10,000 girls.
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According to the National Congenital Anomaly and Rare Diseases Registration Service (NCARDRS), Rett Syndrome is estimated to affect about one in 12,000 girls born each year and is only rarely seen in males.
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As per a study conducted by the Health Programme of the European Union, the estimated prevalence of Rett Syndrome in Europe is 10.0 per 100,000 and the estimated birth prevalence of Rett Syndrome is 5.0 in 100,000.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Rett Syndrome pipeline therapies. It also thoroughly assesses the Rett Syndrome market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the market trend for each marketed Rett Syndrome drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Rett Syndrome Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Rett Syndrome epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Rett Syndrome epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Rett Syndrome Epidemiology, Segmented as –
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Prevalent Cases of Rett Syndrome in the 7MM (2019–2032)
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Gender-specific Prevalent Cases of Rett Syndrome in the 7MM (2019–2032)
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Age-specific Prevalent Cases of Rett Syndrome in the 7MM (2019–2032)
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Diagnosed and Treatable Cases of Rett Syndrome in the 7MM (2019–2032)
Rett Syndrome Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Rett Syndrome market or expected to be launched during the study period. The analysis covers the Rett Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Rett Syndrome drugs based on their sale and 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 Rett Syndrome 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.
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Rett Syndrome Therapeutics Analysis
There are currently no FDA-approved treatments for Rett syndrome. Treatment for the disorder is symptomatic — focusing on the management of symptoms — and supportive, requiring a multidisciplinary approach. Antiepileptic drugs can be used to help control seizures; a class of antidepressants, known as selective serotonin reuptake inhibitors (SSRIs) can be used for the treatment of anxiety; proton pump inhibitors (PPIs) or H2 blockers can help reduce reflux; laxatives can be used to treat constipation.
Several major pharma and biotech companies are developing therapies for Rett Syndrome. Currently, Anavex Life Sciences is leading the therapeutics market with its Rett Syndrome drug candidates in the most advanced stage of clinical development.
Latest Clinical, Commercial, and Regulatory Developments in the Rett Syndrome Therapeutics Market
– On June 15, 2023, DepYmed, Inc. (“DepYmed” or the “Company”) announces the presentation of a development update for its small molecule PTP1B inhibitor program and its lead clinical candidate, DPM-1003, at the 2023 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting.
– On April 17, 2023, Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that DAYBUE™ (trofinetide) is now available for the treatment of Rett syndrome in adult and pediatric patients two years of age and older in the United States. DAYBUE has demonstrated the potential to improve the signs and symptoms of Rett syndrome. DAYBUE was approved by the U.S. Food and Drug Administration (FDA) on March 10, 2023, and is the first and only drug approved by the FDA for the treatment of Rett syndrome.
– On June 12, 2023, Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL) announced the publication of a relevant new peer-reviewed study in the American Journal on Intellectual and Developmental Disabilities, entitled ‘Rett Syndrome Behaviour Questionnaire in Children and Adults With Rett Syndrome: Psychometric Characterization and Revised Factor Structure’.
– On June 15, 2023, Taysha Gene Therapies, Inc. (Nasdaq: TSHA) announced it will host a virtual R&D Day on Wednesday, June 28, 2023, to discuss updates on TSHA-120, a self-complimentary intrathecally delivered investigational AAV9 gene therapy in clinical evaluation for GAN, and TSHA-102, a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome.
Rett Syndrome Companies Actively Working in the Therapeutics Market Include
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Anavex Life Sciences
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Novartis
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Ultragenyx Pharmaceutical
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Q-State Biosciences
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AMO Pharma
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Neurolixis
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Vyant Bio
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Prilenia Therapeutics
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StrideBio
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Taysha Gene Therapies
And Many Others
Emerging and Marketed Rett Syndrome Therapies Covered in the Report Include:
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ANAVEX2-73 (blarcamesine): Anavex Life Sciences
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TSHA-102: Taysha Gene Therapies
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NNZ-2566: Neuren Pharmaceuticals/Acadia Pharmaceuticals
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Dojolvi: Ultragenyx Pharmaceutical
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Rett Syndrome Competitive Intelligence Analysis
4. Rett Syndrome Market Overview at a Glance
5. Rett Syndrome Disease Background and Overview
6. Rett Syndrome Patient Journey
7. Rett Syndrome Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Rett Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Rett Syndrome Unmet Needs
10. Key Endpoints of Rett Syndrome Treatment
11. Rett Syndrome Marketed Therapies
12. Rett Syndrome Emerging Drugs and Latest Therapeutic Advances
13. Rett Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Rett Syndrome Market Outlook (In US, EU5, and Japan)
16. Rett Syndrome Companies Active in the Market
17. Rett Syndrome Access and Reimbursement Overview
18. KOL Views on the Rett Syndrome Market
19. Rett Syndrome Market Drivers
20. Rett Syndrome Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading 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.
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