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Niemann-Pick Disease Type C Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
Report Format: PDF+Excel | Report ID: SR112024A11203
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1 Preface
2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology
3 Executive Summary
4 Niemann-Pick Disease Type C - Introduction
4.1 Overview
4.2 Regulatory Process
4.3 Epidemiology (2018-2023
) and Forecast (2024-2034
)
4.4 Market Overview (2018-2023
) and Forecast (2024-2034
)
4.5 Competitive Intelligence
5 Niemann-Pick Disease Type C - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment
6 Patient Journey
7 Niemann-Pick Disease Type C - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023
)
7.2.2 Epidemiology Forecast (2024-2034
)
7.2.3 Epidemiology by Age (2018-2034)
7.2.4 Epidemiology by Gender (2018-2034)
7.2.5 Diagnosed Cases (2018-2034)
7.2.6 Patient Pool/Treated Cases (2018-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023
)
7.3.2 Epidemiology Forecast (2024-2034
)
7.3.3 Epidemiology by Age (2018-2034)
7.3.4 Epidemiology by Gender (2018-2034)
7.3.5 Diagnosed Cases (2018-2034)
7.3.6 Patient Pool/Treated Cases (2018-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023
)
7.4.2 Epidemiology Forecast (2024-2034
)
7.4.3 Epidemiology by Age (2018-2034)
7.4.4 Epidemiology by Gender (2018-2034)
7.4.5 Diagnosed Cases (2018-2034)
7.4.6 Patient Pool/Treated Cases (2018-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023
)
7.5.2 Epidemiology Forecast (2024-2034
)
7.5.3 Epidemiology by Age (2018-2034)
7.5.4 Epidemiology by Gender (2018-2034)
7.5.5 Diagnosed Cases (2018-2034)
7.5.6 Patient Pool/Treated Cases (2018-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023
)
7.6.2 Epidemiology Forecast (2024-2034
)
7.6.3 Epidemiology by Age (2018-2034)
7.6.4 Epidemiology by Gender (2018-2034)
7.6.5 Diagnosed Cases (2018-2034)
7.6.6 Patient Pool/Treated Cases (2018-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023
)
7.7.2 Epidemiology Forecast (2024-2034
)
7.7.3 Epidemiology by Age (2018-2034)
7.7.4 Epidemiology by Gender (2018-2034)
7.7.5 Diagnosed Cases (2018-2034)
7.7.6 Patient Pool/Treated Cases (2018-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023
)
7.8.2 Epidemiology Forecast (2024-2034
)
7.8.3 Epidemiology by Age (2018-2034)
7.8.4 Epidemiology by Gender (2018-2034)
7.8.5 Diagnosed Cases (2018-2034)
7.8.6 Patient Pool/Treated Cases (2018-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023
)
7.9.2 Epidemiology Forecast (2024-2034
)
7.9.3 Epidemiology by Age (2018-2034)
7.9.4 Epidemiology by Gender (2018-2034)
7.9.5 Diagnosed Cases (2018-2034)
7.9.6 Patient Pool/Treated Cases (2018-2034)
8 Niemann-Pick Disease Type C - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 Niemann-Pick Disease Type C - Unmet Needs
10 Niemann-Pick Disease Type C - Key Endpoints of Treatment
11 Niemann-Pick Disease Type C - Marketed Products
11.1 List of Niemann-Pick Disease Type C Marketed Drugs Across the Top 7 Markets
11.1.1 Zavesca (Miglustat) - Actelion Pharmaceuticals
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets
Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.
12 Niemann-Pick Disease Type C - Pipeline Drugs
12.1 List of Niemann-Pick Disease Type C Pipeline Drugs Across the Top 7 Markets
12.1.1 Trappsol Cyclo - Cyclo Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 VTS-270 - Mandos
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 AZ-3102 - Azafaros
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 Arimoclomol - Zevra Therapeutics
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 Adrabetadex - Mandos
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 Regulatory Status
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report.
13 Niemann-Pick Disease Type C - Attribute Analysis of Key Marketed and Pipeline Drugs
14 Niemann-Pick Disease Type C – Clinical Trial Landscape
14.1 Drugs by Status
14.2 Drugs by Phase
14.3 Drugs by Route of Administration
14.4 Key Regulatory Events
15 Niemann-Pick Disease Type C - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Niemann-Pick Disease Type C - Market Size
15.2.1.1 Market Size (2018-2023
)
15.2.1.2 Market Forecast (2024-2034
)
15.2.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.2.2.1 Market Size by Therapies (2018-2023
)
15.2.2.2 Market Forecast by Therapies (2024-2034
)
15.3 Market Scenario - United States
15.3.1 Niemann-Pick Disease Type C - Market Size
15.3.1.1 Market Size (2018-2023
)
15.3.1.2 Market Forecast (2024-2034
)
15.3.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.3.2.1 Market Size by Therapies (2018-2023
)
15.3.2.2 Market Forecast by Therapies (2024-2034
)
15.3.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Niemann-Pick Disease Type C - Market Size
15.4.1.1 Market Size (2018-2023
)
15.4.1.2 Market Forecast (2024-2034
)
15.4.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.4.2.1 Market Size by Therapies (2018-2023
)
15.4.2.2 Market Forecast by Therapies (2024-2034
)
15.4.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Niemann-Pick Disease Type C - Market Size
15.5.1.1 Market Size (2018-2023
)
15.5.1.2 Market Forecast (2024-2034
)
15.5.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.5.2.1 Market Size by Therapies (2018-2023
)
15.5.2.2 Market Forecast by Therapies (2024-2034
)
15.5.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Niemann-Pick Disease Type C - Market Size
15.6.1.1 Market Size (2018-2023
)
15.6.1.2 Market Forecast (2024-2034
)
15.6.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.6.2.1 Market Size by Therapies (2018-2023
)
15.6.2.2 Market Forecast by Therapies (2024-2034
)
15.6.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Niemann-Pick Disease Type C - Market Size
15.7.1.1 Market Size (2018-2023
)
15.7.1.2 Market Forecast (2024-2034
)
15.7.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.7.2.1 Market Size by Therapies (2018-2023
)
15.7.2.2 Market Forecast by Therapies (2024-2034
)
15.7.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Niemann-Pick Disease Type C - Market Size
15.8.1.1 Market Size (2018-2023
)
15.8.1.2 Market Forecast (2024-2034
)
15.8.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.8.2.1 Market Size by Therapies (2018-2023
)
15.8.2.2 Market Forecast by Therapies (2024-2034
)
15.8.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Niemann-Pick Disease Type C - Market Size
15.9.1.1 Market Size (2018-2023
)
15.9.1.2 Market Forecast (2024-2034
)
15.9.2 Niemann-Pick Disease Type C - Market Size by Therapies
15.9.2.1 Market Size by Therapies (2018-2023
)
15.9.2.2 Market Forecast by Therapies (2024-2034
)
15.9.3 Niemann-Pick Disease Type C - Access and Reimbursement Overview
16 Niemann-Pick Disease Type C - Recent Events and Inputs From Key Opinion Leaders
17 Niemann-Pick Disease Type C Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 Niemann-Pick Disease Type C Market – Strategic Recommendations
19 Appendix
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