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The Morquio syndrome treatment market was valued at USD 96.77 million in 2023, driven by the increased awareness for treatment among healthcare professionals about rare genetic disorders across the globe. The market is expected to grow at a CAGR of 7.35% during the forecast period of 2024-2032, with the values likely to reach USD 191.40 million by 2032.
Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), is a rare genetic disorder caused by deficiencies in enzymes responsible for breaking down glycosaminoglycans (GAGs). This results in the buildup of GAGs in tissues and organs throughout the body, leading to a range of skeletal abnormalities, organ enlargement, and systemic complications.
Currently, ERT is a cornerstone of treatment for Morquio syndrome. It involves infusing patients with the missing enzyme (either GALNS for MPS IVA or beta-galactosidase for MPS IVB), which helps to reduce GAG accumulation and manage symptoms. Moreover, the ongoing research is focused on developing gene therapy, substrate reduction therapy, and other innovative approaches to address the underlying genetic defects more directly. These therapies hold promise for potentially improving outcomes and expanding treatment options and thus driving the market growth.
Advancements in Therapeutic Options
The advancements in therapeutic options play a significant role as a driver for the Morquio syndrome treatment market. The continuous improvements in ERT formulations, dosing schedules, and delivery methods have enhanced the efficacy of treatment for Morquio syndrome. These advancements aim to optimize enzyme delivery, increase enzyme stability, and prolong therapeutic effects, thereby improving patient outcomes. The market involves other emerging therapies also including gene therapy and substrate reduction therapy (SRT). The research into gene therapy aims to address the underlying genetic defect causing Morquio syndrome. SRT targets the accumulation of glycosaminoglycans (GAGs) by reducing their production within cells. This approach aims to slow disease progression and alleviate symptoms, offering an alternative or complementary treatment option to ERT and augmenting the market growth.
Increasing Awareness and Diagnosis is Expected to Propel Morquio Syndrome Treatment Market Demand
The improved awareness among healthcare professionals and the public has led to more accurate and timely diagnoses of Morquio syndrome. As awareness continues to grow, more patients are identified early, which expands the potential market for treatments. For instance, according to an article published by National NPS Society, there is a growing movement promoting newborn screening for MPS. The goals of managing MPS IV include improving quality of life, slowing down the progression of the disease, and prevent permanent tissue and organ damage. Hence, the increasing awareness in diagnosis demands for treatment and is expected to drive the market growth.
The market is witnessing several trends and developments to improve the current global scenario. Some of the notable trends are as follows:
Key Trends | Impact |
Emerging Therapies | Research into novel treatment approaches such as gene therapy, and substrate reduction therapy (SRT) is expanding. These therapies hold promise for addressing underlying genetic defects and reducing the accumulation of glycosaminoglycans (GAGs), potentially offering alternative or complementary treatment options to ERT, and driving the market growth. An article published by National Health Institute reveals that gene therapy for this condition is under investigation. ERT and HSCT in combination with surgical intervention(s) are a therapeutic option for Morquio A. |
Patient-Centric Care and Multidisciplinary Management | There is an increasing emphasis on holistic, multidisciplinary care for Morquio syndrome patients. This includes orthopedic interventions, physical therapy, respiratory support, and management of cardiovascular and other systemic complications. Integrated care approaches aim to improve quality of life and long-term health outcomes. This multidisciplinary approach is expected to drive the market growth in forecast period. |
Market Breakup by Disease Type
Market Breakup by Type of Treatment
Market Breakup by Region
Market Segmentation Based on Type of Treatment is Anticipated to Witness Substantial Growth
By type of treatment, the market is segmented into enzyme replacement therapy and others. The enzyme replacement therapy (ERT) is dominating the market due to its high clinical efficiency. ERT has demonstrated clinical efficacy in managing the symptoms and slowing the progression of Morquio syndrome. It works by replacing the deficient enzyme (either GALNS for MPS IVA or beta-galactosidase for MPS IVB), which helps to break down glycosaminoglycans (GAGs) that accumulate in tissues and organs.
Hence, ERT is growing in the Morquio syndrome treatment market due to its demonstrated clinical efficacy, established safety profile, ongoing advancements in formulations and delivery methods. These factors collectively contribute to the increasing adoption and market penetration of ERT in the management of Morquio syndrome and thus driving the segmental growth.
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Regionally, the market report offers an insight into the United States, EU-4 and the United Kingdom, Germany, France, Italy, Spain, United Kingdom, Japan, and India. The United States, having a robust healthcare infrastructure and a strong presence of prominent pharmaceutical companies, is estimated to hold a high market value in the forecast period. Increasing collaborations among the key market players boost research initiatives that are expected to fuel the region’s market share. The clinical trials and research initiatives are often initiated and conducted in the country, leading to early access to novel therapies for patients leading to regional growth in the market.
Germany holds a significant Morquio syndrome treatment market share due to well-established healthcare infrastructure with advanced medical facilities, specialized treatment centers, and skilled healthcare professionals. This infrastructure supports timely diagnosis, comprehensive management, and access to innovative treatments for rare diseases like Morquio syndrome.
Additionally, Japan is expected to witness substantial market growth due to rising awareness about rare diseases like Morquio syndrome, and educational initiatives. Improved awareness leads to earlier diagnosis and treatment initiation, expanding the market size.
The key features of the market report include patent analysis, clinical trials analysis, grants analysis, funding and investment analysis, and strategic initiatives such as recent partnerships, and collaborations analysis by the leading players. The major companies in the market are as follows:
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. is one of the leading biotechnology companies with their core business and research focus on enzyme replacement therapies. The company was established in 1997 and has its headquarters in California, United States. Their product portfolio consists of treatment solutions and injections related to rare genetic disorders.
JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. is a Japanese biopharmaceutical company known for its focus on developing therapies for rare diseases, including treatments for lysosomal storage disorders like Morquio syndrome (also known as MPS IV).
Other key players in the market include Sanofi, Sangamo Therapeutics, and Alexion Pharmaceuticals Inc. among others.
REPORT FEATURES | DETAILS |
Base Year | 2023 |
Historical Period | 2017-2023 |
Forecast Period | 2024-2032 |
Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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Breakup by Disease Type |
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Breakup by Type of Treatment |
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Breakup by Region |
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Market Dynamics |
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Supplier Landscape |
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Companies Covered |
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
1.4 Research Methodology
2 Executive Summary
3 Morquio Syndrome Treatment Market Overview: 8 Major Market
3.1 Global Morquio Syndrome Treatment Market Historical Value (2017-2023)
3.2 Global Morquio Syndrome Treatment Market Forecast Value (2024-2032)
4 Vendor Positioning Analysis
4.1 Key Vendors
4.2 Prospective Leaders
4.3 Niche Leaders
4.4 Disruptors
5 Morquio Syndrome Treatment: Disease Overview
5.1 Guidelines and Stages
5.2 Pathophysiology
5.3 Screening and Diagnosis
5.4 Therapy Pathway
6 Patient Profile
6.1 Patient Profile Overview
6.2 Patient Psychology and Emotional Impact Factors
6.3 Risk Assessment and Therapy Success Rate
7 Morquio Syndrome Treatment Market Epidemiology Scenario and Forecast – 8 Major Markets
7.1 8MM Morquio Syndrome Treatment Market Epidemiology Scenario Overview (2017-2032)
7.1.1 Prevalence, by Country
7.1.1.1 U.S.
7.1.1.2 U.K.
7.1.1.3 EU4
7.1.1.4 India
7.1.1.5 Japan
7.1.2 Mortality by Country
7.1.2.1 U.S.
7.1.2.2 U.K.
7.1.2.3 EU4
7.1.2.4 India
7.1.2.5 Japan
7.1.3 Treatment Seeking Rate by Country
7.1.3.1 U.S.
7.1.3.2 U.K.
7.1.3.3 EU4
7.1.3.4 India
7.1.3.5 Japan
8 Morquio Syndrome Treatment Market Landscape: 8 Major Market*
8.1 Morquio Syndrome Treatment Market: Developers Landscape
8.1.1 Analysis by Year of Establishment
8.1.2 Analysis by Company Size
8.1.3 Analysis by Region
8.2 Morquio Syndrome Treatment Market: Product Landscape
8.2.1 Analysis by Disease Type
8.2.2 Analysis by Type of Treatment
9 Morquio Syndrome Treatment Market Treatment Challenges and Unmet Needs
9.1 Therapy Pathway Challenges
9.2 Compliance and Drop-Out Analysis
9.3 Awareness and Prevention Gaps
10 Cost of Treatment
11 Morquio Syndrome: Market Dynamics
11.1 Market Drivers and Constraints
11.2 SWOT Analysis
11.2.1 Strengths
11.2.2 Weaknesses
11.2.3 Opportunities
11.2.4 Threats
11.3 PESTEL Analysis
11.3.1 Political
11.3.2 Economic
11.3.3 Social
11.3.4 Technological
11.3.5 Legal
11.3.6 Environment
11.4 Porter’s Five Forces Model
11.4.1 Bargaining Power of Suppliers
11.4.2 Bargaining Power of Buyers
11.4.3 Threat of New Entrants
11.4.4 Threat of Substitutes
11.4.5 Degree of Rivalry
11.5 Key Demand Indicators
11.6 Key Price Indicators
11.7 Industry Events, Initiatives, and Trends
11.8 Value Chain Analysis
12 Morquio Syndrome Treatment Market Segmentation: 8 Major Market
12.1 Morquio Syndrome Treatment Market by Disease Type
12.1.1 Market Overview
12.1.2 Morquio A
12.1.3 Morquio B
12.2 Morquio Syndrome Treatment Market by Type of Treatment
12.2.1 Market Overview
12.2.2 Enzyme Replacement Therapy
12.2.3 Others
12.3 Morquio Syndrome Treatment Market by Region
12.3.1 Market Overview
12.3.2 United States
12.3.3 EU-4 and the United Kingdom
12.3.3.1 Germany
12.3.3.2 France
12.3.3.3 Italy
12.3.3.4 Spain
12.3.3.5 United Kingdom
12.3.4 Japan
12.3.5 India
13 United States Morquio Syndrome Treatment Market (2017-2032)
13.1 United States Morquio Syndrome Treatment Market Historical Value (2017-2023)
13.2 United States Morquio Syndrome Treatment Market Forecast Value (2024-2032)
13.3 United States Morquio Syndrome Treatment Market by Disease Type
13.3.1 Market Overview
13.3.2 Morquio A
13.3.3 Morquio B
13.4 United States Morquio Syndrome Treatment Market by Type of Treatment
13.4.1 Market Overview
13.4.2 Enzyme Replacement Therapy
13.4.3 Others
14 EU-4 and United Kingdom Morquio Syndrome Treatment Market (2017-2032)
14.1 EU-4 and United Kingdom Morquio Syndrome Treatment Market Historical Value (2017-2023)
14.2 EU-4 and United Kingdom Morquio Syndrome Treatment Market Forecast Value (2024-2032)
14.3 EU-4 and United Kingdom Morquio Syndrome Treatment Market by Disease Type
14.3.1 Market Overview
14.3.2 Morquio A
14.3.3 Morquio B
14.4 EU-4 and United Kingdom Morquio Syndrome Treatment Market by Type of Treatment
14.4.1 Market Overview
14.4.2 Enzyme Replacement Therapy
14.4.3 Others
15 Japan Morquio Syndrome Treatment Market
15.1 Japan Morquio Syndrome Treatment Market Historical Value (2017-2023)
15.2 Japan Morquio Syndrome Treatment Market Forecast Value (2024-2032)
15.3 Japan Morquio Syndrome Treatment Market by Disease Type
15.3.1 Market Overview
15.3.2 Morquio A
15.3.3 Morquio B
15.4 Japan Morquio Syndrome Treatment Market by Type of Treatment
15.4.1 Market Overview
15.4.2 Enzyme Replacement Therapy
15.4.3 Others
16 India Morquio Syndrome Treatment Market
16.1 India Morquio Syndrome Treatment Market (2017-2032) Historical Value (2017-2023)
16.2 India Morquio Syndrome Treatment Market (2017-2032) Forecast Value (2024-2032)
16.3 India Morquio Syndrome Treatment Market by Disease Type
16.3.1 Market Overview
16.3.2 Morquio A
16.3.3 Morquio B
16.4 India Morquio Syndrome Treatment Market by Type of Treatment
16.4.1 Market Overview
16.4.2 Enzyme Replacement Therapy
16.4.3 Others
17 Regulatory Framework
17.1 Regulatory Overview
17.2 US FDA
17.3 EU EMA
17.4 INDIA CDSCO
17.5 JAPAN PMDA
17.6 Others
18 Patent Analysis
18.1 Analysis by Type of Patent
18.2 Analysis by Publication Year
18.3 Analysis by Issuing Authority
18.4 Analysis by Patent Age
18.5 Analysis by CPC Analysis
18.6 Analysis by Patent Valuation
18.7 Analysis by Key Players
19 Clinical Trial Analysis
19.1 Analysis by Trial Registration Year
19.2 Analysis by Trial Status
19.3 Analysis by Trial Phase
19.4 Analysis by Therapeutic Area
19.5 Analysis by Geography
20 Grant Analysis
20.1 Analysis by Year
20.2 Analysis by Amount Awarded
20.3 Analysis by Issuing Authority
20.4 Analysis by Grant Product
20.5 Analysis by Funding Institute
20.6 Analysis by Departments
20.7 Analysis by Recipient Organization
21 Funding and Investment Analysis
21.1 Analysis by Funding Instances
21.2 Analysis by Type of Funding
21.3 Analysis by Funding Amount
21.4 Analysis by Leading Players
21.5 Analysis by Leading Investors
21.6 Analysis by Geography
22 Strategic Initiatives
22.1 Analysis by Partnership Instances
22.2 Analysis by Type of Partnership
22.3 Analysis by Leading Players
22.4 Analysis by Geography
23 Supplier Landscape
23.1 Market Share Analysis, By Region (Top 5 Companies)
23.2 BioMarin Pharmaceutical Inc
23.2.1 Financial Analysis
23.2.2 Product Portfolio
23.2.3 Demographic Reach and Achievements
23.2.4 Mergers and Acquisitions
23.2.5 Certifications
23.3 JCR Pharmaceuticals Co., Ltd.
23.3.1 Financial Analysis
23.3.2 Product Portfolio
23.3.3 Demographic Reach and Achievements
23.3.4 Mergers and Acquisitions
23.3.5 Certifications
23.4 Sanofi
23.4.1 Financial Analysis
23.4.2 Product Portfolio
23.4.3 Demographic Reach and Achievements
23.4.4 Mergers and Acquisitions
23.4.5 Certifications
23.5 Sangamo Therapeutics
23.5.1 Financial Analysis
23.5.2 Product Portfolio
23.5.3 Demographic Reach and Achievements
23.5.4 Mergers and Acquisitions
23.5.5 Certifications
23.6 Alexion Pharmaceuticals Inc.
23.6.1 Financial Analysis
23.6.2 Product Portfolio
23.6.3 Demographic Reach and Achievements
23.6.4 Mergers and Acquisitions
23.6.5 Certifications
24 Global Morquio Syndrome Treatment Market – Distribution Model (Additional Insight)
24.1 Overview
24.2 Potential Distributors
24.3 Key Parameters for Distribution Partner Assessment
25 Key Opinion Leaders (KOL) Insights (Additional Insight)
26 Payment Methods (Additional Insight)
26.1 Government Funded
26.2 Private Insurance
26.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
* The coverage of the Market Landscape section depends on the data availability and may cover a minimum of 80% of the total market. The EMR team strives to make this section as comprehensive as possible.
**The supplier list is not exhaustive. Moreover, we can provide analysis of companies as per custom requests.
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