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Sanfilippo Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
Report Format: PDF+Excel | Report ID: SR112024A6735
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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 Sanfilippo Syndrome - Introduction
4.1 Overview
4.2 Epidemiology (2018-2023) and Forecast (2024-2034)
4.3 Market Overview (2018-2023) and Forecast (2024-2034)
4.4 Competitive Intelligence
5 Sanfilippo Syndrome - 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 Sanfilippo Syndrome - 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)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)
8 Sanfilippo Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 Sanfilippo Syndrome - Unmet Needs
10 Sanfilippo Syndrome - Key Endpoints of Treatment
11 Sanfilippo Syndrome - Marketed Products
11.1 List of Sanfilippo Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Drug Name – Company Name
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 complete list of marketed drugs has been provided in the report.
12 Sanfilippo Syndrome - Pipeline Drugs
12.1 List of Sanfilippo Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 Trehalose - Seelos 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 Olenasufligene Relduparvovec - Lysogene
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 Tralesinidase Alpha - BioMarin Pharmaceutical
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 DNL126 - Denali
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 UX 111 - Abeona Therapeutics; Ultragenyx Pharmaceutical
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. Sanfilippo Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs
14 Sanfilippo Syndrome - Market Scenario
14.1 Market Scenario - Key Insights
14.2 Market Scenario - Top 7 Markets
14.2.1 Sanfilippo Syndrome - Market Size
14.2.1.1 Market Size (2018-2023)
14.2.1.2 Market Forecast (2024-2034)
14.2.2 Sanfilippo Syndrome - Market Size by Therapies
14.2.2.1 Market Size by Therapies (2018-2023)
14.2.2.2 Market Forecast by Therapies (2024-2034)
14.3 Market Scenario - United States
14.3.1 Sanfilippo Syndrome - Market Size
14.3.1.1 Market Size (2018-2023)
14.3.1.2 Market Forecast (2024-2034)
14.3.2 Sanfilippo Syndrome - Market Size by Therapies
14.3.2.1 Market Size by Therapies (2018-2023)
14.3.2.2 Market Forecast by Therapies (2024-2034)
14.3.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.4 Market Scenario - Germany
14.4.1 Sanfilippo Syndrome - Market Size
14.4.1.1 Market Size (2018-2023)
14.4.1.2 Market Forecast (2024-2034)
14.4.2 Sanfilippo Syndrome - Market Size by Therapies
14.4.2.1 Market Size by Therapies (2018-2023)
14.4.2.2 Market Forecast by Therapies (2024-2034)
14.4.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.5 Market Scenario - France
14.5.1 Sanfilippo Syndrome - Market Size
14.5.1.1 Market Size (2018-2023)
14.5.1.2 Market Forecast (2024-2034)
14.5.2 Sanfilippo Syndrome - Market Size by Therapies
14.5.2.1 Market Size by Therapies (2018-2023)
14.5.2.2 Market Forecast by Therapies (2024-2034)
14.5.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.6 Market Scenario - United Kingdom
14.6.1 Sanfilippo Syndrome - Market Size
14.6.1.1 Market Size (2018-2023)
14.6.1.2 Market Forecast (2024-2034)
14.6.2 Sanfilippo Syndrome - Market Size by Therapies
14.6.2.1 Market Size by Therapies (2018-2023)
14.6.2.2 Market Forecast by Therapies (2024-2034)
14.6.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.7 Market Scenario - Italy
14.7.1 Sanfilippo Syndrome - Market Size
14.7.1.1 Market Size (2018-2023)
14.7.1.2 Market Forecast (2024-2034)
14.7.2 Sanfilippo Syndrome - Market Size by Therapies
14.7.2.1 Market Size by Therapies (2018-2023)
14.7.2.2 Market Forecast by Therapies (2024-2034)
14.7.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.8 Market Scenario - Spain
14.8.1 Sanfilippo Syndrome - Market Size
14.8.1.1 Market Size (2018-2023)
14.8.1.2 Market Forecast (2024-2034)
14.8.2 Sanfilippo Syndrome - Market Size by Therapies
14.8.2.1 Market Size by Therapies (2018-2023)
14.8.2.2 Market Forecast by Therapies (2024-2034)
14.8.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.9 Market Scenario - Japan
14.9.1 Sanfilippo Syndrome - Market Size
14.9.1.1 Market Size (2018-2023)
14.9.1.2 Market Forecast (2024-2034)
14.9.2 Sanfilippo Syndrome - Market Size by Therapies
14.9.2.1 Market Size by Therapies (2018-2023)
14.9.2.2 Market Forecast by Therapies (2024-2034)
14.9.3 Sanfilippo Syndrome - Access and Reimbursement Overview
15 Sanfilippo Syndrome - Recent Events and Inputs From Key Opinion Leaders
16 Sanfilippo Syndrome Market - SWOT Analysis
16.1 Strengths
16.2 Weaknesses
16.3 Opportunities
16.4 Threats
17 Appendix
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