Summary

The 7 major Morquio syndrome markets are expected to exhibit a CAGR of 4.45% during 2024-2034.

The Morquio syndrome market has been comprehensively analyzed in IMARC's new report titled "Morquio Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), refers to a rare genetic disorder caused by the deficiency or malfunctioning of enzymes involved in the breakdown of certain sugar molecules called glycosaminoglycans (GAGs). The most prominent symptoms associated with the ailment are short stature, skeletal abnormalities, joint laxity, pectus carinatum (protruding chest), hip dysplasia, etc. Various respiratory complications, like obstructive sleep apnea and restrictive lung disease, may also occur. Some other common indications include vision and hearing impairment, cardiac issues, dental problems, etc. The diagnosis of the ailment involves a combination of clinical evaluation, biochemical testing, genetic testing, and imaging studies. Clinical assessment may consist of a physical examination, evaluation of medical history, as well as identification of characteristic symptoms. Several biochemical tests, such as measuring GAGs or enzyme activity levels in blood or urine, aid in confirming the diagnosis. Genetic testing plays a crucial role in identifying specific gene mutations associated with Morquio syndrome. Additionally, numerous imaging studies, including X-rays, CT scans, MRIs, etc., help to visualize skeletal abnormalities and organ involvement.

The increasing cases of deficiency or malfunctioning of specific enzymes, such as N-acetylgalactosamine-6-sulfatase and beta-galactosidase, that disrupt normal tissue functioning are primarily driving the Morquio syndrome market. Furthermore, the rising adoption of effective pain management strategies, including physical therapy, occupational therapy, medications, etc., to help alleviate pain and improve daily functioning in patients is acting as another significant growth-inducing factor. Additionally, the emerging popularity of enzyme replacement therapy (ERT), which involves administering a synthetic form of the deficient enzyme to replace the missing or dysfunctional enzyme responsible for breaking down GAGs, is also creating a positive outlook for the market. Moreover, the increasing utilization of orthopedic interventions, such as corrective surgeries and spinal fusion, for addressing the skeletal issues associated with the ailment, including short stature, skeletal deformities, joint abnormalities, etc., is further augmenting the market growth. Apart from this, the ongoing advancements in biomarker analysis techniques, such as the introduction of liquid chromatography-mass spectrometry (LC-MS) and enzyme-linked immunosorbent assay (ELISA), which have improved the accuracy and sensitivity of biomarker detection, are expected to drive the Morquio syndrome market in the coming years.

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

Morquio 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 Morquio 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 Morquio syndrome market?
What are the key regulatory events related to the Morquio syndrome market?
What is the structure of clinical trial landscape by status related to the Morquio syndrome market?
What is the structure of clinical trial landscape by phase related to the Morquio syndrome market?
What is the structure of clinical trial landscape by route of administration related to the Morquio syndrome market?

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Table of Contents

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 Morquio 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 Morquio 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 Morquio 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 Morquio Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Morquio Syndrome - Unmet Needs

10 Morquio Syndrome - Key Endpoints of Treatment

11 Morquio Syndrome - Marketed Products
11.1 List of Morquio Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Vimizim (Elosulfase alfa) - BioMarin Pharmaceuticals
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 Morquio Syndrome - Pipeline Drugs
12.1 List of Morquio 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. Morquio Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

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

16 Morquio Syndrome - Recent Events and Inputs From Key Opinion Leaders

17 Morquio Syndrome Market - SWOT Analysis
17.1 Strengths
17.2 Weaknesses
17.3 Opportunities
17.4 Threats

18 Morquio Syndrome Market – Strategic Recommendations

19 Appendix