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 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 Acid Sphingomyelinase Deficiency - Unmet Needs
10 Acid Sphingomyelinase Deficiency - Key Endpoints of Treatment
11 Acid Sphingomyelinase Deficiency - Marketed Products
11.1 List of Acid Sphingomyelinase Deficiency Marketed Drugs Across the Top 7 Markets
11.1.1 Xenpozyme (Olipudase alfa) - Sanofi
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 Acid Sphingomyelinase Deficiency - Pipeline Drugs
12.1 List of Acid Sphingomyelinase Deficiency Pipeline Drugs Across the Top 7 Markets
12.1.1 Drug Name – Company Name
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
Kindly note that the complete list of pipeline drugs has been provided in the report.
13. Acid Sphingomyelinase Deficiency - Attribute Analysis of Key Marketed and Pipeline Drugs
14. Acid Sphingomyelinase Deficiency – 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 Acid Sphingomyelinase Deficiency - Market Scenario
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Acid Sphingomyelinase Deficiency - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Acid Sphingomyelinase Deficiency - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Acid Sphingomyelinase Deficiency - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Acid Sphingomyelinase Deficiency - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Acid Sphingomyelinase Deficiency - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Acid Sphingomyelinase Deficiency - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Acid Sphingomyelinase Deficiency - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Acid Sphingomyelinase Deficiency - 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 Acid Sphingomyelinase Deficiency - Access and Reimbursement Overview
16 Acid Sphingomyelinase Deficiency - Recent Events and Inputs From Key Opinion Leaders
17 Acid Sphingomyelinase Deficiency Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 Acid Sphingomyelinase Deficiency Market – Strategic Recommendations
19 Appendix