Mucopolysaccharidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
The 7 major mucopolysaccharidoses markets are expected to exhibit a CAGR of 5.9% during 2024-2034.
The mucopolysaccharidoses market has been comprehensively analyzed in IMARC's new report titled "Mucopolysaccharidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Mucopolysaccharidoses (MPS) refer to a group of rare, inherited metabolic disorders resulting from the absence or malfunctioning of specific enzymes needed to break down molecules called glycosaminoglycans (GAGs). These molecules accumulate in the body's cells, leading to progressive, multi-system damage. Symptoms vary based on the type of MPS but may include developmental delays, skeletal abnormalities, short stature, joint stiffness, coarse facial features, and problems with vision and hearing. Organ systems like the heart, central nervous system, and respiratory system can also be affected, leading to life-threatening complications. Because symptoms are often nonspecific and gradually progressive, diagnosis can be challenging. Typically, physicians may initially suspect MPS based on clinical presentation and may proceed with urine tests to check for elevated GAG levels. A definitive diagnosis is usually made by enzyme assays from blood samples or fibroblasts, and in some cases, genetic testing may be conducted to identify the specific mutation causing the disorder.
The increasing cases of genetic mutations affecting enzymes responsible for breaking down complex sugar molecules, leading to the accumulation of these substances in various tissues and organs, are primarily driving the mucopolysaccharidoses market. In addition to this, the inflating utilization of supportive care measures, including physical and occupational therapies, since they aim to enhance mobility, preserve joint function, and improve overall well-being, is also creating a positive outlook for the market. Moreover, the widespread adoption of hematopoietic stem cell transplantation (HSCT), especially in younger patients with certain types of MPS, is further propelling the market growth. This procedure involves the replacement of defective cells in the bone marrow with healthy ones, thereby correcting the enzyme deficiency. Additionally, the rising usage of efficacious enzyme replacement therapies (ERTs) like laronidase, idursulfase, elosulfase alfa, etc., which help to provide enzymatic activity to reduce the deficient enzymes in individuals suffering from the illness, is acting as another significant growth-inducing factor. Apart from this, the escalating popularity of innovative techniques, such as gene therapy, which focus on the introduction or correction of genetic material within a patient's cells to remedy or counteract the disorder, is expected to drive the mucopolysaccharidoses market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the mucopolysaccharidoses market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for mucopolysaccharidoses and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the mucopolysaccharidoses market in any manner.
Time Period of the Study
Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034
Countries Covered
United States
Germany
France
United Kingdom
Italy
Spain
Japan
Analysis Covered Across Each Country
Historical, current, and future epidemiology scenario
Historical, current, and future performance of the mucopolysaccharidoses market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the mucopolysaccharidoses market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current mucopolysaccharidoses marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
Late-Stage Pipeline Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Key Questions Answered in this Report:
Market Insights
How has the mucopolysaccharidoses market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the mucopolysaccharidoses market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the mucopolysaccharidoses market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?
Epidemiology Insights
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by type across the seven major markets?
How many patients are diagnosed (2018-2034) with mucopolysaccharidoses across the seven major markets?
What is the size of the mucopolysaccharidoses patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of mucopolysaccharidoses?
What will be the growth rate of patients across the seven major markets?
Mucopolysaccharidoses: Current Treatment Scenario, Marketed Drugs and Emerging Therapies
What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for mucopolysaccharidoses drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the mucopolysaccharidoses market?
What are the key regulatory events related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by status related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by phase related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by route of administration related to the mucopolysaccharidoses market?
The mucopolysaccharidoses market has been comprehensively analyzed in IMARC's new report titled "Mucopolysaccharidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Mucopolysaccharidoses (MPS) refer to a group of rare, inherited metabolic disorders resulting from the absence or malfunctioning of specific enzymes needed to break down molecules called glycosaminoglycans (GAGs). These molecules accumulate in the body's cells, leading to progressive, multi-system damage. Symptoms vary based on the type of MPS but may include developmental delays, skeletal abnormalities, short stature, joint stiffness, coarse facial features, and problems with vision and hearing. Organ systems like the heart, central nervous system, and respiratory system can also be affected, leading to life-threatening complications. Because symptoms are often nonspecific and gradually progressive, diagnosis can be challenging. Typically, physicians may initially suspect MPS based on clinical presentation and may proceed with urine tests to check for elevated GAG levels. A definitive diagnosis is usually made by enzyme assays from blood samples or fibroblasts, and in some cases, genetic testing may be conducted to identify the specific mutation causing the disorder.
The increasing cases of genetic mutations affecting enzymes responsible for breaking down complex sugar molecules, leading to the accumulation of these substances in various tissues and organs, are primarily driving the mucopolysaccharidoses market. In addition to this, the inflating utilization of supportive care measures, including physical and occupational therapies, since they aim to enhance mobility, preserve joint function, and improve overall well-being, is also creating a positive outlook for the market. Moreover, the widespread adoption of hematopoietic stem cell transplantation (HSCT), especially in younger patients with certain types of MPS, is further propelling the market growth. This procedure involves the replacement of defective cells in the bone marrow with healthy ones, thereby correcting the enzyme deficiency. Additionally, the rising usage of efficacious enzyme replacement therapies (ERTs) like laronidase, idursulfase, elosulfase alfa, etc., which help to provide enzymatic activity to reduce the deficient enzymes in individuals suffering from the illness, is acting as another significant growth-inducing factor. Apart from this, the escalating popularity of innovative techniques, such as gene therapy, which focus on the introduction or correction of genetic material within a patient's cells to remedy or counteract the disorder, is expected to drive the mucopolysaccharidoses market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the mucopolysaccharidoses market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for mucopolysaccharidoses and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the mucopolysaccharidoses market in any manner.
Time Period of the Study
Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034
Countries Covered
United States
Germany
France
United Kingdom
Italy
Spain
Japan
Analysis Covered Across Each Country
Historical, current, and future epidemiology scenario
Historical, current, and future performance of the mucopolysaccharidoses market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the mucopolysaccharidoses market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current mucopolysaccharidoses marketed drugs and late-stage pipeline drugs.
In-Market Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
Late-Stage Pipeline Drugs
Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance
*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.
Key Questions Answered in this Report:
Market Insights
How has the mucopolysaccharidoses market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the mucopolysaccharidoses market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the mucopolysaccharidoses market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?
Epidemiology Insights
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by gender across the seven major markets?
What is the number of prevalent cases (2018-2034) of mucopolysaccharidoses by type across the seven major markets?
How many patients are diagnosed (2018-2034) with mucopolysaccharidoses across the seven major markets?
What is the size of the mucopolysaccharidoses patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of mucopolysaccharidoses?
What will be the growth rate of patients across the seven major markets?
Mucopolysaccharidoses: Current Treatment Scenario, Marketed Drugs and Emerging Therapies
What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for mucopolysaccharidoses drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the mucopolysaccharidoses market?
What are the key regulatory events related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by status related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by phase related to the mucopolysaccharidoses market?
What is the structure of clinical trial landscape by route of administration related to the mucopolysaccharidoses market?
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 MUCOPOLYSACCHARIDOSES - 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 MUCOPOLYSACCHARIDOSES - 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 MUCOPOLYSACCHARIDOSES - 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 Epidemiology by Type (???2018-2034)
7.2.6 Diagnosed Cases (???2018-2034)
7.2.7 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 Epidemiology by Type (???2018-2034)
7.3.6 Diagnosed Cases (???2018-2034)
7.3.7 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 Epidemiology by Type (???2018-2034)
7.4.6 Diagnosed Cases (???2018-2034)
7.4.7 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 Epidemiology by Type (???2018-2034)
7.5.6 Diagnosed Cases (???2018-2034)
7.5.7 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 Epidemiology by Type (???2018-2034)
7.6.6 Diagnosed Cases (???2018-2034)
7.6.7 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 Epidemiology by Type (???2018-2034)
7.7.6 Diagnosed Cases (???2018-2034)
7.7.7 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 Epidemiology by Type (???2018-2034)
7.8.6 Diagnosed Cases (???2018-2034)
7.8.7 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 Epidemiology by Type (???2018-2034)
7.9.6 Diagnosed Cases (???2018-2034)
7.9.7 Patient Pool/Treated Cases (???2018-2034)
8 MUCOPOLYSACCHARIDOSES - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 MUCOPOLYSACCHARIDOSES - UNMET NEEDS
10 MUCOPOLYSACCHARIDOSES - KEY ENDPOINTS OF TREATMENT
11 MUCOPOLYSACCHARIDOSES - MARKETED PRODUCTS
11.1 List of Mucopolysaccharidoses Marketed Drugs Across the Top 7 Markets
11.1.1 Elaprase (Idursulfase) - Shire Human Genetic Therapies
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
11.1.2 Mepseviitm (Vestronidase Alfa-Vjbk) - Ultragenyx Pharmaceutical
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Vimizim (Elosulfase alfa) - BioMarin International Limited
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets
11.1.4 Aldurazyme (Laronidase) - BioMarin/Sanofi
11.1.4.1 Drug Overview
11.1.4.2 Mechanism of Action
11.1.4.3 Regulatory Status
11.1.4.4 Clinical Trial Results
11.1.4.5 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 MUCOPOLYSACCHARIDOSES - PIPELINE DRUGS
12.1 List of Mucopolysaccharidoses Pipeline Drugs Across the Top 7 Markets
12.1.1 DNL310 - Denali 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 AX 250 - Allievex Corporation
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 ABO-102 - Ultragenyx 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 RGX-121 - Regenxbio
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
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report
13. MUCOPOLYSACCHARIDOSES - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. MUCOPOLYSACCHARIDOSES – 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 MUCOPOLYSACCHARIDOSES - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Mucopolysaccharidoses - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Mucopolysaccharidoses - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Mucopolysaccharidoses - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Mucopolysaccharidoses - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Mucopolysaccharidoses - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Mucopolysaccharidoses - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Mucopolysaccharidoses - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
16 MUCOPOLYSACCHARIDOSES - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 MUCOPOLYSACCHARIDOSES MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 MUCOPOLYSACCHARIDOSES MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
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 MUCOPOLYSACCHARIDOSES - 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 MUCOPOLYSACCHARIDOSES - 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 MUCOPOLYSACCHARIDOSES - 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 Epidemiology by Type (???2018-2034)
7.2.6 Diagnosed Cases (???2018-2034)
7.2.7 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 Epidemiology by Type (???2018-2034)
7.3.6 Diagnosed Cases (???2018-2034)
7.3.7 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 Epidemiology by Type (???2018-2034)
7.4.6 Diagnosed Cases (???2018-2034)
7.4.7 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 Epidemiology by Type (???2018-2034)
7.5.6 Diagnosed Cases (???2018-2034)
7.5.7 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 Epidemiology by Type (???2018-2034)
7.6.6 Diagnosed Cases (???2018-2034)
7.6.7 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 Epidemiology by Type (???2018-2034)
7.7.6 Diagnosed Cases (???2018-2034)
7.7.7 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 Epidemiology by Type (???2018-2034)
7.8.6 Diagnosed Cases (???2018-2034)
7.8.7 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 Epidemiology by Type (???2018-2034)
7.9.6 Diagnosed Cases (???2018-2034)
7.9.7 Patient Pool/Treated Cases (???2018-2034)
8 MUCOPOLYSACCHARIDOSES - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 MUCOPOLYSACCHARIDOSES - UNMET NEEDS
10 MUCOPOLYSACCHARIDOSES - KEY ENDPOINTS OF TREATMENT
11 MUCOPOLYSACCHARIDOSES - MARKETED PRODUCTS
11.1 List of Mucopolysaccharidoses Marketed Drugs Across the Top 7 Markets
11.1.1 Elaprase (Idursulfase) - Shire Human Genetic Therapies
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
11.1.2 Mepseviitm (Vestronidase Alfa-Vjbk) - Ultragenyx Pharmaceutical
11.1.2.1 Drug Overview
11.1.2.2 Mechanism of Action
11.1.2.3 Regulatory Status
11.1.2.4 Clinical Trial Results
11.1.2.5 Sales Across Major Markets
11.1.3 Vimizim (Elosulfase alfa) - BioMarin International Limited
11.1.3.1 Drug Overview
11.1.3.2 Mechanism of Action
11.1.3.3 Regulatory Status
11.1.3.4 Clinical Trial Results
11.1.3.5 Sales Across Major Markets
11.1.4 Aldurazyme (Laronidase) - BioMarin/Sanofi
11.1.4.1 Drug Overview
11.1.4.2 Mechanism of Action
11.1.4.3 Regulatory Status
11.1.4.4 Clinical Trial Results
11.1.4.5 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 MUCOPOLYSACCHARIDOSES - PIPELINE DRUGS
12.1 List of Mucopolysaccharidoses Pipeline Drugs Across the Top 7 Markets
12.1.1 DNL310 - Denali 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 AX 250 - Allievex Corporation
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 ABO-102 - Ultragenyx 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 RGX-121 - Regenxbio
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
Kindly note that the above only represents a partial list of pipeline drugs, and the complete list has been provided in the report
13. MUCOPOLYSACCHARIDOSES - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14. MUCOPOLYSACCHARIDOSES – 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 MUCOPOLYSACCHARIDOSES - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Mucopolysaccharidoses - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Mucopolysaccharidoses - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Mucopolysaccharidoses - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Mucopolysaccharidoses - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Mucopolysaccharidoses - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Mucopolysaccharidoses - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Mucopolysaccharidoses - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Mucopolysaccharidoses - 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 Mucopolysaccharidoses - Access and Reimbursement Overview
16 MUCOPOLYSACCHARIDOSES - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 MUCOPOLYSACCHARIDOSES MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 MUCOPOLYSACCHARIDOSES MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
