Mucopolysaccharidosis III (Sanfilippo syndrome) Pipeline Analysis 2018 - Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Developments.
Mucopolysaccharidosis III (Sanfilippo syndrome) Pipeline Analysis report covers 12 drugs currently in different phases of development. Mucopolysaccharidosis III is a rare inherited autosomal recessive disorder characterised by progressive dementia, aggressive behavior, hyperactivity, seizures, deafness, loss of vision, and an inability to sleep for more than a few hours at a time. The condition causes progressive intellectual disability and the loss of previously acquired skills (developmental regression). The mucopolysaccharidoses III are a rare group of lysosomal storage disorders that are caused by the deficiency or absence of specific lysosomal enzymes that makes the body unable to break down large sugar molecules called glycosaminoglycans (GAG), specifically GAG called heparan sulfate.
The report provides Mucopolysaccharidosis III treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region. The report will help to evaluate the collaboration, in-licensing and out-licensing opportunities, formulating business development strategies and tracking the activities of the key market players. Epidemiology, major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented to offer stakeholders a better understanding of the key factors affecting the overall market environment.
Various databases (for patents and Clinical Trials), studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research.
Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the market.
Major industry players profiled as part of the report are Alexion Pharmaceuticals, Omeros Corporation among others.
Scope:
Mucopolysaccharidosis III (Sanfilippo syndrome) Pipeline Analysis report covers 12 drugs currently in different phases of development. Mucopolysaccharidosis III is a rare inherited autosomal recessive disorder characterised by progressive dementia, aggressive behavior, hyperactivity, seizures, deafness, loss of vision, and an inability to sleep for more than a few hours at a time. The condition causes progressive intellectual disability and the loss of previously acquired skills (developmental regression). The mucopolysaccharidoses III are a rare group of lysosomal storage disorders that are caused by the deficiency or absence of specific lysosomal enzymes that makes the body unable to break down large sugar molecules called glycosaminoglycans (GAG), specifically GAG called heparan sulfate.
The report provides Mucopolysaccharidosis III treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region. The report will help to evaluate the collaboration, in-licensing and out-licensing opportunities, formulating business development strategies and tracking the activities of the key market players. Epidemiology, major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented to offer stakeholders a better understanding of the key factors affecting the overall market environment.
Various databases (for patents and Clinical Trials), studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research.
Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the market.
Major industry players profiled as part of the report are Alexion Pharmaceuticals, Omeros Corporation among others.
Scope:
- By Company
- By Phase
- By Molecule Type
- By Region
- By Route of Administration
Table of Contents
1. Introduction1.1. Research Methodology
1.2. Research Scope
1.2.1. Analysis by Company
1.2.2. Analysis by Phase
1.2.3. By Molecule Type
1.2.4. By region
2. Disease Overview
2.1.1. Introduction
2.1.2. Classification
2.1.3. Symptoms
2.1.4. Causes
2.1.5. Diagnoses
2.1.6. Treatment
2.1.7. Epidemiology
3. Executive Summary
4. Market Dynamics
4.1. Drivers
4.2. Restraints
4.3. Opportunities
5. Pipeline Analysis/Outlook
5.1. Analysis by Company
5.2. Analysis by Phase
5.2.1. Phase of Development
5.2.1.1. Introduction
5.2.1.1.1. Drug Profiling
- Drug Name
- Generic Name
- Synonyms
- Company
- Collaborator
- Route of administration
- Target
- Mechanism of Action
- Technology
- Molecule type
- CAS Number
- Weight
- Chemical Formula
- IUPAC name
- ATC code
5.2.1.1.2. Strategic Developments
5.2.1.1.3. Clinical trials
5.2.1.1.4. Clinical trial results
5.2.1.1.5. Patents
5.2.1.1.6. Technology
5.2.2. Comparative Analysis for Trials by Phase (Pie, Bar graph)
5.3. By Molecule type
5.4. Analysis by Region
6. Company profiling
6.1. BioMarin Pharmaceutical
6.1.1. Introduction
6.1.2. Financials
6.1.3. Products and Services
6.1.4. SWOT
6.2. UniQure Biopharma B.V.
6.2.1. Introduction
6.2.2. Financials
6.2.3. Products and Services
6.2.4. SWOT
6.3. Swedish Orphan Biovitrum
6.3.1. Introduction
6.3.2. Financials
6.3.3. Products and Services
6.3.4. SWOT
6.4. Abeona Therapeutics
6.4.1. Introduction
6.4.2. Financials
6.4.3. Products and Services
6.4.4. SWOT
6.5. Lysogene
6.5.1. Introduction
6.5.2. Financials
6.5.3. Products and Services
6.5.4. SWOT
6.6. Phoenix Nest Inc.
6.6.1. Introduction
6.6.2. Financials
6.6.3. Products and Services
6.6.4. SWOT
6.7. Orchard Therapeutics
6.7.1. Introduction
6.7.2. Financials
6.7.3. Products and Services
6.7.4. SWOT
6.8. ArmaGen
6.8.1. Introduction
6.8.2. Financials
6.8.3. Products and Services
6.8.4. SWOT
6.9. Esteve
6.9.1. Introduction
6.9.2. Financials
6.9.3. Products and Services
6.9.4. SWOT
6.10. Oxford BioMedica Plc
6.10.1. Introduction
6.10.2. Financials
6.10.3. Products and Services
6.10.4. SWOT
6.11. Axcentua
6.11.1. Introduction
6.11.2. Financials
6.11.3. Products and Services
6.11.4. SWOT
Companies Mentioned
- BioMarin Pharmaceutical
- UniQure Biopharma B.V.
- Swedish Orphan Biovitrum
- Abeona Therapeutics
- Lysogene
- Phoenix Nest Inc.
- Orchard Therapeutics
- ArmaGen
- Esteve
- Oxford BioMedica Plc
- Axcentua
Methodology
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