DelveInsight’s Rett syndrome Market Insights report provides a thorough understanding of current treatment practices, emerging Rett syndrome market share of the individual therapies, current and forecasted Rett syndrome market size from 2019 to 2032 segmented into 7MM (the USA, EU5 (the UK, Italy, Spain, France, and Germany), and Japan).
Rett syndrome 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 on 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.
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Some of the key highlights of the Rett Syndrome Market Report
- Through various secondary resources, it can be concluded that Rett Syndrome basically affects females and is rarely seen in males.
- Rett Syndrome is estimated to affect about one in 12,000 girls born each year and is only rarely seen in males.
- Approximately 85-90% of affected people may experience growth failure and muscle wasting that worsens with age.
- Rett Syndrome companies included such as Acadia Pharmaceuticals, GW Research, and several others.
- Rett Syndrome therapies included such as NNZ-2566 (Trofinetide), Epidiolex, and several others.
- Slowed growth. Brain growth slows after birth
- Loss of normal movement and coordination
- Loss of communication abilities
- Abnormal hand movements
- Unusual eye movements
- Breathing problems
- Irritability and crying
- Other abnormal behaviors
Rett Syndrome Causes
Rett syndrome is not a degenerative disorder but causes problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. These can include learning, speech, sensory sensations, mood, movement, breathing, cardiac function, and even chewing, swallowing, and digestion. Rett syndrome is part of a spectrum of disorders with the same genetic cause. Other disorders on the spectrum include PPM-X syndrome, MECP2 duplication syndrome, and MECP2-related severe neonatal encephalopathy. These other conditions can affect males.
Currently, the treatment paradigm for Rett Syndrome includes few treatment options, which are generally supportive in nature such as physical therapy, hydrotherapy, occupational therapy, speech-language therapy and medications and diet management. However, various off-label drugs are used for palliative treatment of Rett Syndrome which include bromocriptine, carbidopa-levodopa, levocarnitine, and certain antidepressants such as trazodone can also be prescribed to ease sleep related problems.
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Rett Syndrome Market Insights
Rett syndrome Treatment is symptomatic, i.e., focusing on the management of symptoms that require a multidisciplinary approach. Treatment basically is categorized into physical therapy, hydrotherapy, occupational therapy, speech-language therapy and medications.
Rett Syndrome Emerging Therapy Assessment
The dynamics of Rett Syndrome market is anticipated to change in the coming years owing to the improvement in the research and development undertaking. There are several key players involved in developing the therapies for Rett Syndrome like Neuren Pharmaceuticals/Acadia Pharmaceuticals (NNZ-2566), GW Research (GWP42003-P), Anavex Life Sciences (Anavex 2-73), Ultragenyx Pharmaceutical (Dojolvi) and others. The launch of emerging therapies is expected during the forecast period of 2021-2030.
Rett Syndrome Market Report Highlights
In more than 99% of people with Rett syndrome, there is no history of the disorder in their family. Many of these cases result from new mutations in the MECP2 gene. More than 200 different mutations have been identified in this gene. This disease is caused by a genetic mutation on the long arm of the X chromosome. These genes cause the brain to develop incorrectly by inappropriately activating other genes in the brain at the wrong time.
Rett Syndrome Epidemiology Insights
- Prevalent Cases
- Gender-specific Prevalent Cases
- Age-specific Prevalent Cases
- Diagnosed and Treatable Cases
Table of content
1. Key Insights
2. Executive Summary of Rett Syndrome
3. Competitive Intelligence Analysis for Rett Syndrome
4. Rett Syndrome: Market Overview at a Glance
5. Rett Syndrome: Disease Background and Overview
6. Patient Journey
7. Rett Syndrome Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Rett Syndrome Unmet Needs
10. Key Endpoints of Rett Syndrome Treatment
11. Rett Syndrome Marketed Products
12. Rett Syndrome Emerging Therapies
13. Rett Syndrome: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Rett Syndrome
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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