Sphingolipidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034
The 7 major sphingolipidoses markets are expected to exhibit a CAGR of 4.01% during 2024-2034.
The sphingolipidoses market has been comprehensively analyzed in IMARC's new report titled "Sphingolipidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Sphingolipidoses are a group of rare inherited metabolic disorders characterized by the abnormal accumulation of sphingolipids, a type of fatty molecule, within cells. These conditions result from deficiencies in specific enzymes responsible for breaking down sphingolipids, leading to their buildup in various tissues throughout the body. The symptoms of the illness can vary widely depending on the specific type of disorder and the organs affected. Common indications include neurological problems, such as developmental delays, seizures, and loss of motor skills. Additionally, patients may experience physical abnormalities, organ enlargement, and a reduced life expectancy. The severity and onset of indications can vary significantly among affected individuals. The diagnosis of sphingolipidoses typically involves a combination of clinical evaluations, family history assessments, and laboratory tests. Healthcare providers may further recommend enzyme activity assays, genetic testing, and imaging studies to confirm the presence of the specific sphingolipid storage disorder among patients.
The escalating prevalence of mutations in specific genes responsible for sphingolipid metabolism, leading to the abnormal buildup of these lipids in cells and tissues throughout the body, is primarily driving the sphingolipidoses market. In addition to this, the inflating utilization of effective therapeutic interventions, such as substrate reduction therapy, that aim to alleviate symptoms and slow disease progression, is also creating a positive outlook for the market. Moreover, the widespread adoption of supportive care measures, including physical and occupational therapies, since they are designed to enhance muscle strength, coordination, and gross motor skills, thereby addressing some of the debilitating aspects of sphingolipidoses, is further bolstering the market growth. Apart from this, the rising usage of enzyme replacement therapies, which involve the intravenous administration of the deficient enzyme to neutralize toxic lipid buildup in individuals suffering from these conditions, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of adjunctive treatments like bone marrow transplantation, that helps promote treatment outcomes, is also augmenting the market growth. Furthermore, the increasing advancements in the field of gene therapy, which offer techniques to insert functional genes into cells and counterbalance or rectify the defective genes responsible for lipid accumulation, are expected to drive the sphingolipidoses market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the sphingolipidoses market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the sphingolipidoses 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 sphingolipidoses across the seven major markets?
What is the number of prevalent cases (2018-2034) of sphingolipidoses by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of sphingolipidoses by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with sphingolipidoses across the seven major markets?
What is the size of the sphingolipidoses 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 sphingolipidoses?
What will be the growth rate of patients across the seven major markets?
Sphingolipidoses: 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 sphingolipidoses 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 sphingolipidoses market?
What are the key regulatory events related to the sphingolipidoses market?
What is the structure of clinical trial landscape by status related to the sphingolipidoses market?
What is the structure of clinical trial landscape by phase related to the sphingolipidoses market?
What is the structure of clinical trial landscape by route of administration related to the sphingolipidoses market?
The sphingolipidoses market has been comprehensively analyzed in IMARC's new report titled "Sphingolipidoses Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Sphingolipidoses are a group of rare inherited metabolic disorders characterized by the abnormal accumulation of sphingolipids, a type of fatty molecule, within cells. These conditions result from deficiencies in specific enzymes responsible for breaking down sphingolipids, leading to their buildup in various tissues throughout the body. The symptoms of the illness can vary widely depending on the specific type of disorder and the organs affected. Common indications include neurological problems, such as developmental delays, seizures, and loss of motor skills. Additionally, patients may experience physical abnormalities, organ enlargement, and a reduced life expectancy. The severity and onset of indications can vary significantly among affected individuals. The diagnosis of sphingolipidoses typically involves a combination of clinical evaluations, family history assessments, and laboratory tests. Healthcare providers may further recommend enzyme activity assays, genetic testing, and imaging studies to confirm the presence of the specific sphingolipid storage disorder among patients.
The escalating prevalence of mutations in specific genes responsible for sphingolipid metabolism, leading to the abnormal buildup of these lipids in cells and tissues throughout the body, is primarily driving the sphingolipidoses market. In addition to this, the inflating utilization of effective therapeutic interventions, such as substrate reduction therapy, that aim to alleviate symptoms and slow disease progression, is also creating a positive outlook for the market. Moreover, the widespread adoption of supportive care measures, including physical and occupational therapies, since they are designed to enhance muscle strength, coordination, and gross motor skills, thereby addressing some of the debilitating aspects of sphingolipidoses, is further bolstering the market growth. Apart from this, the rising usage of enzyme replacement therapies, which involve the intravenous administration of the deficient enzyme to neutralize toxic lipid buildup in individuals suffering from these conditions, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of adjunctive treatments like bone marrow transplantation, that helps promote treatment outcomes, is also augmenting the market growth. Furthermore, the increasing advancements in the field of gene therapy, which offer techniques to insert functional genes into cells and counterbalance or rectify the defective genes responsible for lipid accumulation, are expected to drive the sphingolipidoses market during the forecast period.
IMARC Group's new report provides an exhaustive analysis of the sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the sphingolipidoses market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current sphingolipidoses 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 sphingolipidoses 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 sphingolipidoses market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the sphingolipidoses 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 sphingolipidoses across the seven major markets?
What is the number of prevalent cases (2018-2034) of sphingolipidoses by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of sphingolipidoses by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with sphingolipidoses across the seven major markets?
What is the size of the sphingolipidoses 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 sphingolipidoses?
What will be the growth rate of patients across the seven major markets?
Sphingolipidoses: 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 sphingolipidoses 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 sphingolipidoses market?
What are the key regulatory events related to the sphingolipidoses market?
What is the structure of clinical trial landscape by status related to the sphingolipidoses market?
What is the structure of clinical trial landscape by phase related to the sphingolipidoses market?
What is the structure of clinical trial landscape by route of administration related to the sphingolipidoses 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 SPHINGOLIPIDOSES - UNMET NEEDS
10 SPHINGOLIPIDOSES - KEY ENDPOINTS OF TREATMENT
11 SPHINGOLIPIDOSES - MARKETED PRODUCTS
11.1 List of Sphingolipidoses Marketed Drugs Across the Top 7 Markets
11.1.1 Galafold (Migalastat) - Amicus Therapeutics
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 Elfabrio (Pegunigalsidase Alfa-Iwxj) - Chiesi Farmaceutici
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 Cerezyme (Imiglucerase) - Genzyme Corporation
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 Vpriv (Velaglucerase Alfa For Injection) - Shire Human Genetic Therapies
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
11.1.5 Elelyso (Taliglucerase Alfa) - Pfizer/Protalix Biotherapeutics
11.1.5.1 Drug Overview
11.1.5.2 Mechanism of Action
11.1.5.3 Regulatory Status
11.1.5.4 Clinical Trial Results
11.1.5.5 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 SPHINGOLIPIDOSES - PIPELINE DRUGS
12.1 List of Sphingolipidoses Pipeline Drugs Across the Top 7 Markets
12.1.1 LY3884961 - Prevail 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 ST-920 - Sangamo Therapeutics
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 Arimoclomol - ZevraDenmark
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 4D-310 - 4D Molecular 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 TAK-611 - Takeda
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 SPHINGOLIPIDOSES - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14 SPHINGOLIPIDOSES – 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 SPHINGOLIPIDOSES - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Sphingolipidoses - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Sphingolipidoses - 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 Sphingolipidoses - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Sphingolipidoses - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Sphingolipidoses - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Sphingolipidoses - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Sphingolipidoses - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Sphingolipidoses - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Sphingolipidoses - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
16 SPHINGOLIPIDOSES - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 SPHINGOLIPIDOSES MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 SPHINGOLIPIDOSES 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - 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 SPHINGOLIPIDOSES - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm
9 SPHINGOLIPIDOSES - UNMET NEEDS
10 SPHINGOLIPIDOSES - KEY ENDPOINTS OF TREATMENT
11 SPHINGOLIPIDOSES - MARKETED PRODUCTS
11.1 List of Sphingolipidoses Marketed Drugs Across the Top 7 Markets
11.1.1 Galafold (Migalastat) - Amicus Therapeutics
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 Elfabrio (Pegunigalsidase Alfa-Iwxj) - Chiesi Farmaceutici
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 Cerezyme (Imiglucerase) - Genzyme Corporation
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 Vpriv (Velaglucerase Alfa For Injection) - Shire Human Genetic Therapies
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
11.1.5 Elelyso (Taliglucerase Alfa) - Pfizer/Protalix Biotherapeutics
11.1.5.1 Drug Overview
11.1.5.2 Mechanism of Action
11.1.5.3 Regulatory Status
11.1.5.4 Clinical Trial Results
11.1.5.5 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 SPHINGOLIPIDOSES - PIPELINE DRUGS
12.1 List of Sphingolipidoses Pipeline Drugs Across the Top 7 Markets
12.1.1 LY3884961 - Prevail 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 ST-920 - Sangamo Therapeutics
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 Arimoclomol - ZevraDenmark
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 4D-310 - 4D Molecular 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 TAK-611 - Takeda
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 SPHINGOLIPIDOSES - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS
14 SPHINGOLIPIDOSES – 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 SPHINGOLIPIDOSES - MARKET SCENARIO
15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
15.2.1 Sphingolipidoses - Market Size
15.2.1.1 Market Size (2018-2023)
15.2.1.2 Market Forecast (2024-2034)
15.2.2 Sphingolipidoses - 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 Sphingolipidoses - Market Size
15.3.1.1 Market Size (2018-2023)
15.3.1.2 Market Forecast (2024-2034)
15.3.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.4 Market Scenario - Germany
15.4.1 Sphingolipidoses - Market Size
15.4.1.1 Market Size (2018-2023)
15.4.1.2 Market Forecast (2024-2034)
15.4.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.5 Market Scenario - France
15.5.1 Sphingolipidoses - Market Size
15.5.1.1 Market Size (2018-2023)
15.5.1.2 Market Forecast (2024-2034)
15.5.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
15.6.1 Sphingolipidoses - Market Size
15.6.1.1 Market Size (2018-2023)
15.6.1.2 Market Forecast (2024-2034)
15.6.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.7 Market Scenario - Italy
15.7.1 Sphingolipidoses - Market Size
15.7.1.1 Market Size (2018-2023)
15.7.1.2 Market Forecast (2024-2034)
15.7.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.8 Market Scenario - Spain
15.8.1 Sphingolipidoses - Market Size
15.8.1.1 Market Size (2018-2023)
15.8.1.2 Market Forecast (2024-2034)
15.8.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
15.9 Market Scenario - Japan
15.9.1 Sphingolipidoses - Market Size
15.9.1.1 Market Size (2018-2023)
15.9.1.2 Market Forecast (2024-2034)
15.9.2 Sphingolipidoses - 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 Sphingolipidoses - Access and Reimbursement Overview
16 SPHINGOLIPIDOSES - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS
17 SPHINGOLIPIDOSES MARKET - SWOT ANALYSIS
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats
18 SPHINGOLIPIDOSES MARKET – STRATEGIC RECOMMENDATIONS
19 APPENDIX
