Reports

Global Morquio Syndrome Treatment Market to Grow at a CAGR of 5.70% During 2024-2032, Driven by the growing investment in research and development (R&D) in the pharmaceutical sector.

Sale

Morquio Syndrome Treatment Market

Morquio Syndrome Treatment Market Size, Share, Trends: By Disease Type: Morquio A, Morquio B; By Type of Treatment: Enzyme Replacement Therapy, Others; Regional Analysis; Market Dynamics: SWOT Analysis, PESTEL Analysis, Porter’s Five Forces Model, Key Demand Indicators, Key Price Indicators; Supplier Landscape; 2024-2032

Morquio Syndrome Treatment Market Size

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 Treatment Market Outlook

  • The advancement in treatment such as introduction and ongoing development of ERT (Enzyme Replacement Therapy) have significantly improved the management of Morquio syndrome. Ongoing research into gene therapy, substrate reduction therapy, and other novel approaches are expanding the treatment landscape.
  • The comprehensive management of Morquio syndrome involves a multidisciplinary approach that includes orthopedic interventions, physical therapy, respiratory support, and management of systemic complications such as cardiovascular issues. The demand for supportive care services is bolstering the overall market for Morquio syndrome treatment.
  • United States is expected to lead the market share driven by increased investments in research initiatives and a well-established healthcare infrastructure.

 

Morquio Syndrome Treatment Market Overview

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.

 

Morquio Syndrome Treatment Market Growth Drivers

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.

 

Morquio Syndrome Treatment Market Trends

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.

 

Morquio Syndrome Treatment Market Segmentation

Market Breakup by Disease Type
     

  • Morquio A
  • Morquio B

 

Market Breakup by Type of Treatment

  • Enzyme Replacement Therapy
  • Others

 

Market Breakup by Region

  • United States
  • EU-4 and the United Kingdom
    • Germany
    • France
    • Italy
    • Spain
    • United Kingdom
  • Japan
  • India

 

Morquio Syndrome Treatment Market Share

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.

 

morquio syndrome treatment market by region

Read more about this report - REQUEST FREE SAMPLE COPY IN PDF

 

Morquio Syndrome Treatment Market Analysis by Region

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.

 

Leading Players in the Morquio Syndrome Treatment Market

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.

 

Key Highlights of the Report

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:

  • Disease Type
  • Type of Treatment
  • Region
Breakup by Disease Type
  • Morquio A
  • Morquio B
Breakup by Type of Treatment
  • Enzyme Replacement Therapy
  • Others 
Breakup by Region
  • United States
  • EU-4 and the United Kingdom
  • Japan
  • India
Market Dynamics
  • Market Drivers and Constraints
  • SWOT Analysis
  • Porter’s Five Forces Model 
  • Key Demand Indicators
  • Key Price Indicators
  • Industry Events, Initiatives, and Trends 
  • Value Chain Analysis
Supplier Landscape
  • Market Structure
  • Company Profiles
    • Financial Analysis
    • Product Portfolio
    • Demographic Reach and Achievements
    • Mergers and Acquisitions
    • Certifications
Companies Covered
  • BioMarin Pharmaceutical Inc 
  • JCR Pharmaceuticals Co., Ltd.
  • Sanofi
  • Sangamo Therapeutics
  • Alexion Pharmaceuticals Inc.

 

Key Questions Answered in the Morquio Syndrome Treatment Market Report

  • What was the Morquio syndrome treatment market value in 2023?
  • What is the Morquio syndrome treatment market forecast outlook for 2024-2032?
  • What are the regional markets covered in the EMR report?
  • What is market segmentation based on disease type?
  • What is market segmentation based on type of treatment?
  • What are the major factors aiding the Morquio syndrome treatment market demand? 
  • How has the market performed so far and how is it anticipated to perform in the coming years?
  • What are the major trends influencing the market? 
  • What are the market's major drivers, opportunities, and restraints? 
  • Which regional market is expected to lead the market share in the forecast period? 
  • Which country is expected to experience expedited growth during the forecast period? 
  • How does the prevalence of genetic disorders affect the market landscape?
  • How does the growing technological advancements in treatment impact the market size?
  • Who are the key players involved in the Morquio syndrome treatment market?
  • What are the current unmet needs and challenges in the market?
  • How are partnerships, collaborations, mergers and acquisitions among the key market players shaping the market dynamics?

 

*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.

Purchase Full Report

Datasheet

$ 2969     $2699
  • Selected Sections, One User
  • Printing Not Allowed
  • Email Delivery in PDF
  • Free Limited Customisation
  • Post Sales Analyst Support
  • 50% Discount on Next Update
Buy Now

Single User License

$ 5499     $4999
  • All Sections, One User
  • One Print Allowed
  • Email Delivery in PDF
  • Free Limited Customisation
  • Post Sales Analyst Support
  • 50% Discount on Next Update
Buy Now
most popular

Five User License

$ 6599     $5999
  • All Sections, Five Users
  • Five Prints Allowed
  • Email Delivery in PDF
  • Free Limited Customisation
  • Post Sales Analyst Support
  • 50% Discount on Next Update
Buy Now

Corporate License

$ 7699     $6999
  • All Sections, Unlimited Users
  • Unlimited Prints Allowed
  • Email Delivery in PDF + Excel
  • Free Limited Customisation
  • Post Sales Analyst Support
  • 50% Discount on Next Update
Buy Now

Any Question? Speak With An Analyst

Contact Analyst

View A Sample

Request Sample

Did You Miss Anything, Ask Now

Request Customisation
Why Expert Market Research?

Right People

We are technically excellent, strategic, practical, experienced and efficient; our analysts are hand-picked based on having the right attributes to work successfully and execute projects based on your expectations.

Right Methodology

We leverage our cutting-edge technology, our access to trusted databases, and our knowledge of the current models used in the market to deliver you research solutions that are tailored to your needs and put you ahead of the curve.

Right Price

We deliver in-depth and superior quality research in prices that are reasonable, unmatchable, and shows our understanding of your resource structure. We, additionally, offer attractive discounts on our upcoming reports.

Right Support

Our team of expert analysts are at your beck and call to deliver you optimum results that are customised to meet your precise needs within the specified timeframe and help you form a better understanding of the industry.

NEWSLETTER