GM2 Gangliosidoses – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030
GM2 Gangliosidoses Disease overview, pathogenesis, identified and emerging biomarkers, pipeline assessment, competitive landscape, KOLs perspective, Clinical Trial Landscaping, Probable launch date estimation Regulatory framework, Epidemiology; Incidence, Prevalence, and current therapy market share, Order of entry, patient share, Market uptake, Peak patient share, Disease burden, Annual cost of therapy, the market forecast for the USA, Germany, France, Italy, Spain, UK, Japan and China 2020 to 2030
- 1. GM2 Gangliosidoses – Key Findings Summary
- 1.1. Clinical findings
- 1.1.1. Disease overview
- 1.1.2. Therapeutic practices
- 1.1.3. Future outlook
- 1.2. Commercial findings
- 1.2.1. GM2 Gangliosidoses market scenario 2020
- 1.2.2. GM2 Gangliosidoses market scenario 2025
- 1.2.3. GM2 Gangliosidoses market scenario 2030
- 2. GM2 Gangliosidoses Overview
- 2.1. Disease Introduction
- 2.2. Pathophysiology
- 2.3. Signs and Symptoms
- 2.4. Risk Factors
- 2.5. Etiology
- 2.6. Classification
- 2.7. Pathogenesis
- 2.8. Diagnosis
- 2.9. Complications
- 2.10. Treatment Algorithm
- 2.10.1. Treatment in US (guidelines)
- 2.10.2. Treatment in EU-5 (guidelines)
- 2.10.3. Treatment in Japan (guidelines)
- 2.10.4. Treatment in China (guidelines)
- 2.11. Treatment Goals for GM2 Gangliosidoses
- 2.12. Referral Patterns
- 2.12.1. Referral Scenario in US
- 2.12.2. Referral Scenario in EU-5
- 2.12.3. Referral Scenario in Japan
- 2.12.4. Referral Scenario in China
- 2.13.GM2 Gangliosidoses Prognosis
- 2.14.Healthcare burden
- 2.14.1. Healthcare burden in US
- 2.14.2. Healthcare burden in EU-5
- 2.14.3. Healthcare burden in Japan
- 2.14.4. Healthcare burden in China
- 2.15. Unmet Needs in GM2 Gangliosidoses management
- 2.16. Market Opportunity for GM2 Gangliosidoses
- 2.17. KOL Comments on current and upcoming/expected treatment practices in GM2 Gangliosidoses
- 3. Epidemiology
- 3.1. Epidemiology Overview
- 3.2. Epidemiology by Geography
- 3.2.1. GM2 Gangliosidoses Epidemiology in US (2019-2030)
- 3.2.1.1. Incidence of GM2 Gangliosidoses
- 3.2.1.2. Diagnosed cases
- 3.2.1.3. Treatable Patient Pool
- 3.2.1.4. Epidemiology Trends
- 3.2.2. GM2 Gangliosidoses Epidemiology in EU-5 (2019-2030)
- 3.2.2.1. Incidence of GM2 Gangliosidoses
- 3.2.2.2. Diagnosed cases
- 3.2.2.3. Treatable Patient Pool
- 3.2.2.4. Epidemiology Trends
- 3.2.3. GM2 Gangliosidoses Epidemiology in Japan (2019-2030)
- 3.2.3.1. Incidence of GM2 Gangliosidoses
- 3.2.3.2. Diagnosed cases
- 3.2.3.3. Treatable Patient Pool
- 3.2.3.4. Epidemiology Trends
- 3.2.4. GM2 Gangliosidoses Epidemiology in China (2019-2030)
- 3.2.4.1. Incidence of GM2 Gangliosidoses
- 3.2.4.2. Diagnosed cases
- 3.2.4.3. Treatable Patient Pool
- 3.2.4.4. Epidemiology Trends
- 3.3. Epidemiology Trends (World-wide)
- 4. Market Outlook
- 4.1. US GM2 Gangliosidoses Market Forecast 2019-2030
- 4.1.1. Market Progression (Futuristic)
- 4.1.2. Market Trends and Expectations
- 4.1.2.1. Worst case scenario
- 4.1.2.2. Base Case Scenario
- 4.1.2.3. Best Case Scenario
- 4.1.3. Drivers and Barriers
- 4.2. UK GM2 Gangliosidoses Market Forecast 2019-2030
- 4.2.1. Market Progression (Futuristic)
- 4.2.2. Market Trends and Expectations
- 4.2.2.1. Worst case scenario
- 4.2.2.2. Base Case Scenario
- 4.2.2.3. Best Case Scenario
- 4.2.3. Drivers and Barriers
- 4.3. France GM2 Gangliosidoses Market Forecast 2019-2030
- 4.3.1. Market Progression (Futuristic)
- 4.3.2. Market Trends and Expectations
- 4.3.2.1. Worst case scenario
- 4.3.2.2. Base Case Scenario
- 4.3.2.3. Best Case Scenario
- 4.3.3. Drivers and Barriers
- 4.4. Germany GM2 Gangliosidoses Market Forecast 2019-2030
- 4.4.1. Market Progression (Futuristic)
- 4.4.2. Market Trends and Expectations
- 4.4.2.1. Worst case scenario
- 4.4.2.2. Base Case Scenario
- 4.4.2.3. Best Case Scenario
- 4.4.3. Drivers and Barriers
- 4.5. Italy GM2 Gangliosidoses Market Forecast 2019-2030
- 4.5.1. Market Progression (Futuristic)
- 4.5.2. Market Trends and Expectations
- 4.5.2.1. Worst case scenario
- 4.5.2.2. Base Case Scenario
- 4.5.2.3. Best Case Scenario
- 4.5.3. Drivers and Barriers
- 4.6. Spain GM2 Gangliosidoses Market Forecast 2019-2030
- 4.6.1. Market Progression (Futuristic)
- 4.6.2. Market Trends and Expectations
- 4.6.2.1. Worst case scenario
- 4.6.2.2. Base Case Scenario
- 4.6.2.3. Best Case Scenario
- 4.6.3. Drivers and Barriers
- 4.7. Japan GM2 Gangliosidoses Market Forecast 2019-2030
- 4.7.1. Market Progression (Futuristic)
- 4.7.2. Market Trends and Expectations
- 4.7.2.1. Worst case scenario
- 4.7.2.2. Base Case Scenario
- 4.7.2.3. Best Case Scenario
- 4.7.3. Drivers and Barriers
- 4.8. China GM2 Gangliosidoses Market Forecast 2019-2030
- 4.8.1. Market Progression (Futuristic)
- 4.8.2. Market Trends and Expectations
- 4.8.2.1. Worst case scenario
- 4.8.2.2. Base Case Scenario
- 4.8.2.3. Best Case Scenario
- 4.8.3. Drivers and Barriers
- 4.9. Key Expected Milestones (world-wide) Impacting the Market
- 5. Competitive Landscape
- 5.1. Pipeline Therapies Overview
- 5.1.1. Phase III Therapies
- 5.1.1.1. Current Status
- 5.1.1.2. Trial details, results
- 5.1.1.3. Approval Timeline
- 5.1.1.4. Likelihood of approval
- 5.1.1.5. Expected Product Positioning
- 5.1.1.2. All other Phase III Therapies …..
- 5.1.1.3. Attribute Analysis of Phase III molecules
- 5.1.2. Phase II and Phase I/II Therapies
- 5.1.2.1. Current Status
- 5.1.2.2. Trial details, results
- 5.1.2.3. Approval Timelines
- 5.1.3. List of active Pre-clinical Therapies
- 5.1.3.1. Status in GM2 Gangliosidoses
- 5.1.3.2. Company positioning
- 5.1.3.2. All other pre-clinical therapies
- 5.1.4. List of Inactive/discontinued assets
- 5.1.4.1. Business impact of discontinuations on current pipeline
- 5.1.5. Potential winners from GM2 Gangliosidoses Pipeline
- 5.1.5.1. Potential Blockbusters across the pipeline
- 6. Regulatory/Approval Scenario
- 6.1. Regulatory/Approval Framework in US
- 6.1.1. Policy Framework
- 6.1.2. Payer Expectations
- 6.2. Regulatory/Approval Framework in UK
- 6.2.1. Policy Framework
- 6.2.2. Payer Expectations
- 6.3. Regulatory/Approval Framework in France
- 6.3.1. Policy Framework
- 6.3.2. Payer Expectations
- 6.4. Regulatory/Approval Framework in Germany
- 6.4.1. Policy Framework
- 6.4.2. Payer Expectations
- 6.5. Regulatory/Approval Framework in Italy
- 6.5.1. Policy Framework
- 6.5.2. Payer Expectations
- 6.6. Regulatory/Approval Framework in Spain
- 6.6.1. Policy Framework
- 6.6.2. Payer Expectations
- 6.7. Regulatory/Approval Framework in Japan
- 6.7.1. Policy Framework
- 6.7.2. Payer Expectations
- 6.8. Regulatory/Approval Framework in China
- 6.8.1. Policy Framework
- 6.8.2. Payer Expectations
- 7. Clinical Trial Assessment – Current and Future Paradigm
- 7.1. Distribution of Primary Endpoints across trials
- 7.2. Distribution of Secondary Endpoints across trials
- 7.3. Evolution and acceptance of surrogate endpoints
- 7.4. Key Investigator initiated trials
- 7.5. Attrition analysis
- 7.5.1. Suspended/Discontinued Assets
- 7.5.2. Failed Trials, Reasons and Business Impact
- 7.5.3. Terminated Trials, Reasons and Business Impact
- 7.5.4. Withdrawn Trials, Reasons and Business Impact
- 7.6. Trial enrollment scenario and challenges
- 7.7. Clinical Trial Guidance (across geographies)
- 8. Thelansis Commentary
- 8.1. Key Unmet needs in GM2 Gangliosidoses
- 8.2. Possible Best-case Clinical Trial Strategies
- 8.3. Possible Best Case Targeted Product Profile (TPP)
- 8.4. Possible Best-case Market positioning strategies
- 8.5. Possible Best-case Market Access Strategies
- 8.6. Possible Best-case LCM Strategies
- 8.7. Overall View on GM2 Gangliosidoses Market in Dollar Value
- 9. Report Methodology
- 9.1. Secondary research
- 9.2. Primary research
- 9.3. Data collation
- 9.4. Insight Generation
- 10. About Thelansis
- 10.1. Our Capabilities
- 10.2. Our Services
- 10.3. Our Contacts
- 10.4. Disclaimer
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