
Mucopolysaccharidosis III - Pipeline Insight, 2024
Description
Mucopolysaccharidosis III - Pipeline Insight, 2024
DelveInsight’s, “Mucopolysaccharidosis III – Pipeline Insight, 2024,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Mucopolysaccharidosis III 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
Mucopolysaccharidosis III: Overview
Mucopolysaccharidosis type III (MPS III) is a multisystem lysosomal storage disease characterized by progressive central nervous system degeneration manifest as severe intellectual disability (ID), developmental regression, and other neurologic manifestations including autism spectrum disorder (ASD), behavioral problems, and sleep disturbances. Systemic manifestations can include musculoskeletal problems (joint stiffness, contractures, scoliosis, and hip dysplasia), hearing loss, respiratory tract and sinopulmonary infections, and cardiac disease (valvular thickening, defects in the cardiac conduction system). Children with MPS IIIA do not produce enough enzyme activity to break down a substance in the body called heparan sulfate. The lack of enzyme activity is due to a defective gene called N-sulfoglucosamine sulfohydrolase (SGSH). To diagnose MPS III, mucopolysaccharides are usually first measured in urine, followed by measurement of enzyme activity in blood or a small skin sample. Increased heparan sulfate in urine, and a decrease in the activity of any one of the four enzymes (shown in the table above) is usually consistent with a diagnosis of MPS III and will identify the MPS III type (A, B, C or D). Treatment of Sanfilippo syndrome is symptomatic and supportive.
""Mucopolysaccharidosis III - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Mucopolysaccharidosis III pipeline landscape is provided which includes the disease overview and Mucopolysaccharidosis III treatment guidelines. The assessment part of the report embraces, in depth Mucopolysaccharidosis III 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, Mucopolysaccharidosis III 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 Mucopolysaccharidosis III R&D. The therapies under development are focused on novel approaches to treat/improve Mucopolysaccharidosis III.
This segment of the Mucopolysaccharidosis III 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.
Mucopolysaccharidosis III Emerging Drugs
- LYS-SAF302: Lysogene
- Tralesinidase alfa: Allievex Corporation
Further product details are provided in the report……..
Mucopolysaccharidosis III: Therapeutic Assessment
This segment of the report provides insights about the different Mucopolysaccharidosis III drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Mucopolysaccharidosis III
- 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
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Mucopolysaccharidosis III: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Mucopolysaccharidosis III 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 Mucopolysaccharidosis III drugs.
Mucopolysaccharidosis III Report Insights
- Mucopolysaccharidosis III 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 Mucopolysaccharidosis III drugs?
- How many Mucopolysaccharidosis III drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Mucopolysaccharidosis III?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Mucopolysaccharidosis III 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 Mucopolysaccharidosis III and their status?
- What are the key designations that have been granted to the emerging drugs?
Table of Contents
50 Pages
- Introduction
- Executive Summary
- Mucopolysaccharidosis III: Overview
- Causes
- Mechanism of Action
- 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
- Mucopolysaccharidosis III – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Mucopolysaccharidosis III companies’ collaborations, Licensing, Acquisition -Deal Value Trends
- Mucopolysaccharidosis III Collaboration Deals
- Company-Company Collaborations (Licensing / Partnering) Analysis
- Company-University Collaborations (Licensing / Partnering) Analysis
- Late Stage Products (Phase III)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II/III)
- Comparative Analysis
- LYS-SAF302: Lysogene
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early stage products (Phase I/II)
- Comparative Analysis
- ABO-102: Abeona Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Mucopolysaccharidosis III Key Companies
- Mucopolysaccharidosis III Key Products
- Mucopolysaccharidosis III- Unmet Needs
- Mucopolysaccharidosis III- Market Drivers and Barriers
- Mucopolysaccharidosis III- Future Perspectives and Conclusion
- Mucopolysaccharidosis III Analyst Views
- Mucopolysaccharidosis III Key Companies
- Appendix
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