
Rett Syndrome - Pipeline Insight, 2024
Description
Rett Syndrome - Pipeline Insight, 2024
DelveInsight’s, “Rett Syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Rett Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Rett Syndrome: Overview
Rett syndrome is a rare genetic neurological disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat, and even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements. Rett syndrome is usually recognized in children between 6 to 18 months as they begin to miss developmental milestones or lose abilities they had gained. Rett syndrome is caused by mutations on the X chromosome on a gene called MECP2. There are more than 900 different mutations found on the MECP2 gene. Most of these mutations are found in eight different “hot spots.” Rett syndrome is not a degenerative disorder with individuals living to middle age or beyond. Rett syndrome occurs worldwide in 1 of every 10,000 female births, and is even rarer in boys. Rett syndrome can present with a wide range of disability ranging from mild to severe. The course and severity of Rett syndrome is determined by the location, type and severity of the mutation and X-inactivation.
The main signs and symptoms of ret syndrome includes slowed growth loss of movement and coordination activities, loss of communication abilities and unusual hand movements, unusual eye movements, breathing problems, irritability and crying, other unusual behaviors, intellectual disabilities, seizures, sideways curvature of the spine (scoliosis), irregular heartbeat, sleep disturbances.
Diagnosing Rett syndrome involves careful observation of the child's growth and development and answering questions about medical and family history. The diagnosis is usually considered when slowing of head growth is noticed or loss of skills or developmental milestones occurs. Rett syndrome after evaluation, genetic testing (DNA analysis) may be needed to confirm the diagnosis other tests are CT scan, EEG (electroencephalogram), Genetic testing, MRI these all tests are involved in diagnosing the syndrome and helpful in the further treatment of the disease.
Although there is no cure for rett syndrome, treatments address symptoms and provide support. These may improve the potential for movement, communication and social participation. Treatments that can help children and adults with rett syndrome include, regular medical care, medications, physical therapy, occupational therapy, speech-language therapy, nutritional support, behavioral intervention, support services. Early intervention programs and school, social and job-training services may help with integration into school, work and social activities special adaptations may make participation possible.
""Rett Syndrome - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Rett Syndrome pipeline landscape is provided which includes the disease overview and Rett Syndrome treatment guidelines. The assessment part of the report embraces, in depth Rett Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Rett Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Rett Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Rett Syndrome.
This segment of the Rett Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Rett Syndrome Emerging Drugs
- Blarcamesine: Anavex Life Sciences Corp
- Vatiquinone: PTC Therapeutics
- TSHA-102: Taysha GTx
Further product details are provided in the report……..
Rett Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Rett Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Rett Syndrome
- There are approx. 15+ key companies which are developing the therapies for Rett Syndrome. The companies which have their Rett Syndrome drug candidates in the most advanced stage, i.e. phase III include, Anavex Life Sciences Corp.
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Rett Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Rett Syndrome therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Rett Syndrome drugs.
Rett Syndrome Report Insights
- Rett Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Rett Syndrome drugs?
- How many Rett Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Rett Syndrome?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Rett Syndrome therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Rett Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
- Blarcamesine
- TSHA-102
- Vatiquinone
- NGN-401
- GXV 001
- Cannabidivarin
- AMO 04
- Anavex Life Sciences Corp.
- Taysha Gene Therapies, Inc.
- PTC Therapeutics
- Neurogene Inc.
- GEXVal
- GW Pharmaceuticals
- AMO Pharma
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Rett Syndrome : Overview
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Rett Syndrome – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Blarcamesine: Anavex Life Sciences Corp
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Vatiquinone: PTC Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- TSHA-102: Taysha GTx
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Rett Syndrome Key Companies
- Rett Syndrome Key Products
- Rett Syndrome - Unmet Needs
- Rett Syndrome - Market Drivers and Barriers
- Rett Syndrome - Future Perspectives and Conclusion
- Rett Syndrome Analyst Views
- Rett Syndrome Key Companies
- Appendix
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