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Sly Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034

May 2024 | 135 pages | ID: S1960EA9CD46EN
IMARC Group

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The 7 major Sly syndrome markets reached a value of US$ 670.1 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 934.7 Million by 2034, exhibiting a growth rate (CAGR) of 3.07% during 2024-2034.

The Sly syndrome market has been comprehensively analyzed in IMARC's new report titled "Sly Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Sly syndrome, also known as Mucopolysaccharidosis type VII (MPS VII), is a rare and severe inherited metabolic disorder characterized by the deficiency of the enzyme beta-glucuronidase. This enzyme is responsible for breaking down glycosaminoglycans (GAGs) in the body. In Sly syndrome, the absence of functional beta-glucuronidase leads to the progressive accumulation of GAGs in various tissues and organs, resulting in widespread cellular damage and dysfunction. The symptoms of Sly syndrome typically appear in early infancy or childhood and vary in severity. Some of the common clinical manifestations include skeletal abnormalities, such as coarse facial features, joint stiffness, skeletal dysplasia, etc. Various other symptoms may involve hepatosplenomegaly (enlargement of the liver and spleen), cardiomyopathy (heart muscle disease), respiratory issues, and neurological complications like developmental delay, intellectual disability, and behavioral problems. The diagnosis of Sly syndrome involves a combination of clinical evaluation, biochemical testing, and genetic analysis. A physical examination may reveal the characteristic facial features and skeletal abnormalities. Blood and urine tests can detect elevated levels of GAGs, pointing to the presence of mucopolysaccharidosis. Further confirmation is achieved through enzyme activity testing, where the deficiency of beta-glucuronidase is established. Genetic testing is vital for identifying specific mutations in the GUSB gene, which confirms the diagnosis of Sly syndrome and helps in providing accurate genetic counseling for affected families.

The increasing prevalence of mutations in the GUSB gene, which result in the deficiency of the enzyme beta-glucuronidase, is primarily driving the Sly syndrome market. In addition to this, the expanding utilization of enzyme replacement therapy (ERT), a treatment that involves administering the missing enzyme intravenously, is also creating a positive outlook for the market. ERT aims to replace the deficient enzyme and alleviate the build-up of GAGs in affected tissues, mitigating disease progression and improving patients' quality of life. Furthermore, the widespread adoption of supportive therapies, such as physical and occupational therapies, to address musculoskeletal and neurological issues is also contributing to the market growth. These therapies help in enhancing muscle strength, improving coordination, and enhancing gross motor skills in affected individuals. Apart from this, ongoing advancements in gene therapy, a promising approach involving the introduction of functional genetic material to correct the mutated GUSB gene, hold potential for transformative outcomes in the treatment of Sly syndrome, further driving the market growth during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the Sly syndrome 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 Sly syndrome 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 Sly syndrome 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 Sly syndrome market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the Sly syndrome market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current Sly syndrome 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 Sly syndrome 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 Sly syndrome market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the Sly syndrome 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 Sly syndrome across the seven major markets?
What is the number of prevalent cases (2018-2034) of Sly syndrome by age across the seven major markets?
What is the number of prevalent cases (2018-2034) of Sly syndrome by gender across the seven major markets?
How many patients are diagnosed (2018-2034) with Sly syndrome across the seven major markets?
What is the size of the Sly syndrome 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 Sly syndrome?
What will be the growth rate of patients across the seven major markets?

Sly Syndrome: 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 Sly syndrome 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 Sly syndrome market?
What are the key regulatory events related to the Sly syndrome market?
What is the structure of clinical trial landscape by status related to the Sly syndrome market?
What is the structure of clinical trial landscape by phase related to the Sly syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Sly syndrome 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 SLY SYNDROME - 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 SLY 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 SLY 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?)
  7.2.3 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 SLY SYNDROME - TREATMENT ALGORITHM, GUIDELINES, AND MEDICAL PRACTICES

8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 SLY SYNDROME - UNMET NEEDS

10 SLY SYNDROME - KEY ENDPOINTS OF TREATMENT

11 SLY SYNDROME - MARKETED PRODUCTS

11.1 List of Sly Syndrome Marketed Drugs Across the Top 7 Markets
  11.1.1 Mepsevii (Vestronidase alfa) - Ultragenyx Pharmaceutical
    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 Market
Kindly note that the above only represents a partial list of marketed drugs, and the complete list has been provided in the report.

12 SLY SYNDROME - PIPELINE DRUGS

12.1 List of Sly Syndrome 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. SLY SYNDROME - ATTRIBUTE ANALYSIS OF KEY MARKETED AND PIPELINE DRUGS

14. SLY SYNDROME – 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 SLY SYNDROME - MARKET SCENARIO

15.1 Market Scenario - Key Insights
15.2 Market Scenario - Top 7 Markets
  15.2.1 Sly Syndrome - Market Size
    15.2.1.1 Market Size (2018-2023)
    15.2.1.2 Market Forecast (?2024-2034?)
  15.2.2 Sly Syndrome - 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 Sly Syndrome - Market Size
    15.3.1.1 Market Size (2018-2023)
    15.3.1.2 Market Forecast (?2024-2034?)
  15.3.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.4 Market Scenario - Germany
  15.4.1 Sly Syndrome - Market Size
    15.4.1.1 Market Size (2018-2023)
    15.4.1.2 Market Forecast (?2024-2034?)
  15.4.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.5 Market Scenario - France
  15.5.1 Sly Syndrome - Market Size
    15.5.1.1 Market Size (2018-2023)
    15.5.1.2 Market Forecast (?2024-2034?)
  15.5.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.6 Market Scenario - United Kingdom
  15.6.1 Sly Syndrome - Market Size
    15.6.1.1 Market Size (2018-2023)
    15.6.1.2 Market Forecast (?2024-2034?)
  15.6.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.7 Market Scenario - Italy
  15.7.1 Sly Syndrome - Market Size
    15.7.1.1 Market Size (2018-2023)
    15.7.1.2 Market Forecast (?2024-2034?)
  15.7.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.8 Market Scenario - Spain
  15.8.1 Sly Syndrome - Market Size
    15.8.1.1 Market Size (2018-2023)
    15.8.1.2 Market Forecast (?2024-2034?)
  15.8.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview
15.9 Market Scenario - Japan
  15.9.1 Sly Syndrome - Market Size
    15.9.1.1 Market Size (2018-2023)
    15.9.1.2 Market Forecast (?2024-2034?)
  15.9.2 Sly Syndrome - 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 Sly Syndrome - Access and Reimbursement Overview

16 SLY SYNDROME - RECENT EVENTS AND INPUTS FROM KEY OPINION LEADERS

17 SLY SYNDROME MARKET - SWOT ANALYSIS

17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 SLY SYNDROME MARKET – STRATEGIC RECOMMENDATIONS

19 APPENDIX


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