Tuberous Sclerosis- Pipeline Insight, 2025
Description
DelveInsight’s, “Tuberous Sclerosis- Pipeline Insight, 2025” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Tuberous Sclerosis 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
Tuberous Sclerosis: Overview
Tuberous sclerosis, also known as tuberous sclerosis complex, is a rare genetic condition that causes mainly non-cancerous (benign) tumors to develop in different parts of the body. The tumors most often affect the brain, skin, kidneys, heart, eyes and lungs. Tuberous sclerosis is present from birth, although it may not cause obvious problems immediately. Tuberous sclerosis is caused by changes (mutations) in either the TSC1 or TSC2 gene. These genes are involved in regulating cell growth, and the mutations lead to uncontrolled growth and multiple tumors throughout the body.
The common symptoms of the disease are caused by noncancerous growths in parts of the body, most commonly in the skin, brain, eyes, kidneys, heart and lungs. But any part of the body can be affected. Symptoms can range from mild to severe, depending on the size or location of the growths. Although symptoms are different for each person with tuberous sclerosis, they can include skin changes, seizures, problems in thinking, reasoning and learning, behavior problems, kidney problems, heart issues, lung problems eye problems and dental changes.
Depending on symptoms, the child may see several different specialists who are experts in tuberous sclerosis. These may include specialists in problems of the brain (neurologist), heart (cardiologist), eyes (ophthalmologist), skin (dermatologist) and kidneys (nephrologist). The health care provider usually does a physical exam and talks with about symptoms and family history. The provider looks for growths, also called noncancerous tumors that are commonly caused by tuberous sclerosis. The provider also will likely order several tests — including blood tests and genetic tests — to diagnose tuberous sclerosis and identify related problems. Seizure evaluation diagnostic testing will likely include an electroencephalogram sometimes called an EEG. Brain, lungs, kidneys and liver evaluation to detect growths in the body, tests may include MRI, CT scan, and Ultrasound and heart evaluation to determine whether the heart is affected, tests usually include echocardiogram and electrocardiogram. Eye exam and dental exam developmental or mental health evaluation genetic testing.
Although there is no cure for tuberous sclerosis, treatment can help manage specific symptoms. For example medicine anti-seizure medicines may be prescribed to control seizures, surgery, therapy, educational and vocational services, mental health and behavior issues. Tuberous sclerosis is a lifelong condition that requires careful ongoing monitoring and follow-up because many symptoms may take years to develop.
""Tuberous Sclerosis- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tuberous Sclerosis pipeline landscape is provided which includes the disease overview and Tuberous Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Tuberous Sclerosis 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, Tuberous Sclerosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Tuberous Sclerosis 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.
Tuberous Sclerosis Emerging Drugs
Further product details are provided in the report……..
Tuberous Sclerosis: Therapeutic Assessment
This segment of the report provides insights about the different Tuberous Sclerosis drugs segregated based on following parameters that define the scope of the report, such as:
Tuberous Sclerosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Tuberous Sclerosis 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 Tuberous Sclerosis drugs.
Tuberous Sclerosis Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Tuberous Sclerosis: Overview
Tuberous sclerosis, also known as tuberous sclerosis complex, is a rare genetic condition that causes mainly non-cancerous (benign) tumors to develop in different parts of the body. The tumors most often affect the brain, skin, kidneys, heart, eyes and lungs. Tuberous sclerosis is present from birth, although it may not cause obvious problems immediately. Tuberous sclerosis is caused by changes (mutations) in either the TSC1 or TSC2 gene. These genes are involved in regulating cell growth, and the mutations lead to uncontrolled growth and multiple tumors throughout the body.
The common symptoms of the disease are caused by noncancerous growths in parts of the body, most commonly in the skin, brain, eyes, kidneys, heart and lungs. But any part of the body can be affected. Symptoms can range from mild to severe, depending on the size or location of the growths. Although symptoms are different for each person with tuberous sclerosis, they can include skin changes, seizures, problems in thinking, reasoning and learning, behavior problems, kidney problems, heart issues, lung problems eye problems and dental changes.
Depending on symptoms, the child may see several different specialists who are experts in tuberous sclerosis. These may include specialists in problems of the brain (neurologist), heart (cardiologist), eyes (ophthalmologist), skin (dermatologist) and kidneys (nephrologist). The health care provider usually does a physical exam and talks with about symptoms and family history. The provider looks for growths, also called noncancerous tumors that are commonly caused by tuberous sclerosis. The provider also will likely order several tests — including blood tests and genetic tests — to diagnose tuberous sclerosis and identify related problems. Seizure evaluation diagnostic testing will likely include an electroencephalogram sometimes called an EEG. Brain, lungs, kidneys and liver evaluation to detect growths in the body, tests may include MRI, CT scan, and Ultrasound and heart evaluation to determine whether the heart is affected, tests usually include echocardiogram and electrocardiogram. Eye exam and dental exam developmental or mental health evaluation genetic testing.
Although there is no cure for tuberous sclerosis, treatment can help manage specific symptoms. For example medicine anti-seizure medicines may be prescribed to control seizures, surgery, therapy, educational and vocational services, mental health and behavior issues. Tuberous sclerosis is a lifelong condition that requires careful ongoing monitoring and follow-up because many symptoms may take years to develop.
""Tuberous Sclerosis- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tuberous Sclerosis pipeline landscape is provided which includes the disease overview and Tuberous Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Tuberous Sclerosis 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, Tuberous Sclerosis 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 Tuberous Sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Tuberous Sclerosis.
This segment of the Tuberous Sclerosis 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.
Tuberous Sclerosis Emerging Drugs
- Ganaxolone: Marinus Pharmaceuticals
- Basimglurant: Noema Pharma AG
Further product details are provided in the report……..
Tuberous Sclerosis: Therapeutic Assessment
This segment of the report provides insights about the different Tuberous Sclerosis drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Tuberous Sclerosis
- There are approx. 8+ key companies which are developing the therapies for Tuberous Sclerosis. The companies which have their Tuberous Sclerosis drug candidates in the most advanced stage, i.e. phase III include, Marinus Pharmaceuticals.
- 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
Tuberous Sclerosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Tuberous Sclerosis 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 Tuberous Sclerosis drugs.
Tuberous Sclerosis Report Insights
- Tuberous Sclerosis 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 Tuberous Sclerosis drugs?
- How many Tuberous Sclerosis drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Tuberous Sclerosis?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Tuberous Sclerosis 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 Tuberous Sclerosis and their status?
- What are the key designations that have been granted to the emerging drugs?
- Marinus Pharmaceuticals
- Virpax Pharmaceuticals
- Noema Pharma
- BridgeBio Pharma
- Ganaxolone
- Cannabidiol
- Basimglurant
- BBP 821
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Tuberous Sclerosis: 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
- Tuberous Sclerosis– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Ganaxolone: Marinus Pharmaceuticals, Inc.
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Basimglurant: Noema Pharma
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- BBP 821: BridgeBio Pharma
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Tuberous Sclerosis Key Companies
- Tuberous Sclerosis Key Products
- Tuberous Sclerosis- Unmet Needs
- Tuberous Sclerosis- Market Drivers and Barriers
- Tuberous Sclerosis- Future Perspectives and Conclusion
- Tuberous Sclerosis Analyst Views
- Tuberous Sclerosis Key Companies
- Appendix
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