Global Morquio Syndrome Treatment Market Size By Type of Treatment, By Mode of Administration, By End-User, By Geographic Scope And Forecast

Report ID: 388262|No. of Pages: 178

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Global Morquio Syndrome Treatment Market Size By Type of Treatment, By Mode of Administration, By End-User, By Geographic Scope And Forecast

Report ID: 388262|Published Date: Feb 2024|No. of Pages: 178|Base Year for Estimate: 2023|Format:   Report available in PDF formatReport available in Excel Format

Morquio Syndrome Treatment Market Size And Forecast

Morquio Syndrome Treatment Market size was valued at USD 155 Million in 2023 and is projected to reach USD 305 Million by 2030, growing at a CAGR of 13.9% during the forecast period 2024-2030.

Morquio Syndrome Treatment Market is estimated to grow at a CAGR of 13.9% & reach USD 305 Mn by the end of 2030

Global Morquio Syndrome Treatment Market Drivers

The market drivers for the Morquio Syndrome Treatment Market can be influenced by various factors. These may include:

  • Research developments: The development of new treatment modalities and targeted therapeutics for Morquio syndrome is aided by ongoing research developments in the fields of genetics, biomarker discovery, and therapeutic interventions.
  • Enhanced Diagnosis and Awareness: As medical professionals, patients, and caregivers become more knowledgeable of the signs and symptoms of Morquio syndrome, as well as the diagnostic standards and treatment choices that are available, the disease can be better managed through early detection, prompt intervention, and therapy.
  • Orphan Drug Designations: Pharmaceutical companies are encouraged to invest in the research of novel therapeutics for rare disorders like Morquio syndrome, as well as orphan drug designations, fast-track approvals, and market exclusivity, among other regulatory incentives.
  • Increasing Healthcare Expenditure: Access to treatments for Morquio syndrome is made easier by increased healthcare spending, government financing for research on rare diseases, and reimbursement support for orphan pharmaceuticals. These factors also improve patient affordability and the uptake of therapeutic interventions in the market.
  • Collaborative Research Initiatives: These efforts, along with public-private partnerships and academic-industry collaborations, stimulate innovation and pipeline development by fostering stakeholder collaboration, speeding up drug discovery, and facilitating clinical trials for treatments for Morquio syndrome.
  • Better Screening and Diagnosis Tools: Developments in diagnostic technology, such as genetic testing, enzyme assays, and imaging modalities, allow for more precise diagnosis, disease tracking, and patient stratification for customized treatment plans for Morquio syndrome.
  • Patient Advocacy and Support Groups: Patient organizations, support groups, and non-profit foundations engage in advocacy work to increase public awareness, supply resources, and push for financing for research, changes to regulations, and patient access to supportive care services and therapies for Morquio syndrome.
  • Gene Therapy and Enzyme Replacement: By treating the underlying enzyme deficit, lowering the severity of the condition, and enhancing patients’ quality of life, new gene therapy techniques and enzyme replacement therapies (ERTs) present encouraging treatment possibilities for Morquio syndrome.
  • Regulatory Approvals and Market Expansion: Across the globe, the market’s reach is increased, patients with Morquio syndrome have more treatment options, and therapeutic outcomes are improved through regulatory approvals of new medicines, orphan drug designations, and extended indications for current treatments.
  • Improvement in Patient Outcomes: Patients with Morquio syndrome experience improved survival rates, slower disease progression, and more functional capacity as a result of treatment advancements, supportive care practices, and multidisciplinary management strategies.

Global Morquio Syndrome Treatment Market Restraints

Several factors can act as restraints or challenges for the Morquio Syndrome Treatment Market. These may include:

  • Restricted Treatment choices: Access to effective interventions and the best possible management of the disease is hampered by the limited availability of approved medications and treatment choices for Morquio syndrome, especially in areas with resource restrictions and healthcare inequities.
  • Exorbitant Treatment Costs: Patients, caregivers, and healthcare systems have financial difficulties due to the exorbitant costs of orphan medications, enzyme replacement therapy, and supportive care interventions. This creates barriers to access and affordability for treatments for Morquio syndrome.
  • Clinical Trial Difficulties: Research on rare diseases, such as Morquio syndrome, faces difficulties in recruiting patients, carrying out clinical trials, and meeting meaningful objectives. These difficulties lead to a lack of clinical data, protracted drug development schedules, and regulatory obstacles for novel treatments.
  • Disease Complexity and Heterogeneity: The multisystem involvement, unpredictable disease progression, and heterogeneity of Morquio syndrome present difficulties for patient stratification, personalized medicine methods, and treatment development. These factors necessitate the implementation of comprehensive disease management techniques.
  • Lack of Disease Awareness: Insufficient knowledge of the signs, diagnostic standards, and management guidelines of Morquio syndrome among medical professionals, general practitioners, and specialists causes underreporting, delays diagnosis, and increases the risk of diagnostic errors and treatment delays.
  • Unmet Medical Needs: There is a continuing need for novel research, funding, and cooperative efforts to discover treatments for Morquio syndrome. These needs include the absence of cures, disease-modifying medications, and symptomatic relief choices.
  • Adverse Effects and Safety Concerns: Treatment adherence, patient monitoring, and risk management are made more difficult by the potential adverse effects, immunogenicity, and long-term safety risks connected to gene therapy vectors, enzyme replacement therapies, and new treatments for Morquio syndrome.
  • Limited Healthcare Infrastructure: Access to comprehensive diagnostic, treatment, and supportive care services for patients with Morquio syndrome is restricted by inadequate healthcare infrastructure, specialized care centers, and multidisciplinary care teams in underserved regions. This exacerbates disparities in the delivery of healthcare.
  • Regulatory and Reimbursement Barriers: In emerging markets and low-income nations, in particular, regulatory complexities, orphan drug designation requirements, and reimbursement difficulties for rare disease therapies obstruct market entry, commercialization, and patient access to treatments for Morquio syndrome.
  • Prolonged Approval and Development Timelines: Protracted market approval procedures, regulatory review procedures, and market approval delays for orphan medications and treatments for uncommon diseases extend the time to market, raise development expenses, and restrict patient access to novel therapeutics for Morquio syndrome.

Global Morquio Syndrome Treatment Market Segmentation Analysis

The Global Morquio Syndrome Treatment Market is Segmented on the basis of Type of Treatment, Mode of Administration, End-User, and Geography.

Morquio Syndrome Treatment Market Segments Analysis

By Type of Treatment:

  • Enzyme Replacement Therapy (ERT): Treatment involving the administration of exogenous enzyme replacement to compensate for the deficient enzyme activity in Morquio syndrome patients, aimed at reducing substrate accumulation and improving symptoms.
  • Surgical Interventions: Surgical procedures such as spinal decompression, cervical spine fusion, and orthopedic surgeries to address skeletal abnormalities, spinal cord compression, and joint deformities associated with Morquio syndrome.
  • Pain Management: Pharmacological interventions, physical therapy, and supportive care measures for managing pain, discomfort, and mobility issues in Morquio syndrome patients, including nonsteroidal anti-inflammatory drugs (NSAIDs) and opioid analgesics.
  • Physical and Occupational Therapy: Rehabilitation programs, physical exercises, and adaptive equipment interventions to improve mobility, muscle strength, joint flexibility, and activities of daily living (ADLs) in Morquio syndrome patients.

By Mode of Administration:

  • Intravenous (IV) Infusion: Enzyme replacement therapy administered via intravenous infusion, typically in a clinical setting, to deliver the recombinant enzyme directly into the bloodstream for systemic distribution and uptake by target tissues and organs.
  • Oral Administration: Oral medications, supplements, and pain relievers administered via the oral route for managing symptoms such as pain, inflammation, and gastrointestinal issues associated with Morquio syndrome.
  • Intra-Articular Injection: Intra-articular injections of corticosteroids, hyaluronic acid, or analgesics into affected joints to alleviate pain, inflammation, and stiffness associated with joint abnormalities and degenerative changes in Morquio syndrome patients.

By End-User:

  • Hospitals and Clinics: Treatment facilities, hospitals, specialized clinics, and healthcare centers providing diagnostic, therapeutic, and rehabilitativeservices for Morquio syndrome patients, including enzyme replacement therapy, surgical interventions, and supportive care.
  • Specialty Centers: Specialized centers, academic institutions, and research hospitals offering comprehensive multidisciplinary care, genetic counseling, clinical trials, and innovative therapies for rare genetic disorders such as Morquio syndrome.
  • Home Healthcare: Home healthcare services, nursing care, and palliative care programs providing personalized treatment, monitoring, and support for Morquio syndrome patients in home settings, including home infusion therapy and rehabilitation services.
  • Rehabilitation Centers: Rehabilitation facilities, physical therapy centers, and orthopedic clinics offering rehabilitative programs, adaptive equipment, and mobility aids for Morquio syndrome patients to improve functional independence and quality of life.

By Geography:

  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East & Africa

Global Morquio Syndrome Treatment Market Keyplayers

The major players in the Morquio Syndrome Treatment Market are:

  • BioMarin Pharmaceuticals Inc. (USA)
  • Ultragenyx Pharmaceutical Inc. (USA)
  • Shire plc (Ireland)
  • ArmaGen, Inc. (USA)
  • Enzyvant Therapeutics Inc. (USA)
  • Amicus Therapeutics (US)
  • Orchard Therapeutics (US)
  • Spark Therapeutics (US)
  • MyoGene Inc. (US)
  • Abeona Therapeutics Inc. (US)
  • Dimension Therapeutics Inc. (US)

Report Scope

REPORT ATTRIBUTESDETAILS
STUDY PERIOD

2020-2030

BASE YEAR

2023

FORECAST PERIOD

2024-2030

HISTORICAL PERIOD

2020-2022

UNIT

Value (USD Million)

KEY COMPANIES PROFILED

BioMarin Pharmaceuticals Inc. (USA), Ultragenyx Pharmaceutical Inc. (USA), Shire plc (Ireland), ArmaGen, Inc. (USA), Enzyvant Therapeutics Inc. (USA), Amicus Therapeutics (US), Orchard Therapeutics (US), Spark Therapeutics (US), MyoGene Inc. (US), Abeona Therapeutics Inc. (US), Dimension Therapeutics Inc. (US)

SEGMENTS COVERED

Type of Treatment, Mode of Administration, End-User, and Geography.

CUSTOMIZATION SCOPE

Free report customization (equivalent up to 4 analyst’s working days) with purchase. Addition or alteration to country, regional & segment scope

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Research Methodology of Verified Market Research:

Research Methodology of VMR

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Reasons to Purchase this Report

• Qualitative and quantitative analysis of the market based on segmentation involving both economic as well as non-economic factors
• Provision of market value (USD Billion) data for each segment and sub-segment
• Indicates the region and segment that is expected to witness the fastest growth as well as to dominate the market
• Analysis by geography highlighting the consumption of the product/service in the region as well as indicating the factors that are affecting the market within each region
• Competitive landscape which incorporates the market ranking of the major players, along with new service/product launches, partnerships, business expansions and acquisitions in the past five years of companies profiled
• Extensive company profiles comprising of company overview, company insights, product benchmarking and SWOT analysis for the major market players
• The current as well as the future market outlook of the industry with respect to recent developments (which involve growth opportunities and drivers as well as challenges and restraints of both emerging as well as developed regions
• Includes an in-depth analysis of the market of various perspectives through Porter’s five forces analysis
• Provides insight into the market through Value Chain
• Market dynamics scenario, along with growth opportunities of the market in the years to come
• 6-month post-sales analyst support

Customization of the Report

• In case of any Queries or Customization Requirements please connect with our sales team, who will ensure that your requirements are met.

Frequently Asked Questions

Morquio Syndrome Treatment Market size was valued at USD 155 Million in 2023 and is projected to reach USD 305 Million by 2030, growing at a CAGR of 13.9% during the forecast period 2024-2030.

The Morquio Syndrome Treatment Market is primarily driven by advancements in medical research, increasing awareness and diagnosis of rare genetic disorders, and the development of novel therapies targeting the underlying causes of the disease.

The major players in the Morquio Syndrome Treatment Market are BioMarin Pharmaceuticals Inc. (USA), Ultragenyx Pharmaceutical Inc. (USA), Shire plc (Ireland), ArmaGen, Inc. (USA), Enzyvant Therapeutics Inc. (USA), Amicus Therapeutics (US), Orchard Therapeutics (US), Spark Therapeutics (US), MyoGene Inc. (US), Abeona Therapeutics Inc. (US), Dimension Therapeutics Inc. (US) Etc

The Global Morquio Syndrome Treatment Market is Segmented on the basis of Type of Treatment, Mode of Administration, End-User, and Geography.

The sample report for the Morquio Syndrome Treatment Market can be obtained on demand from the website. Also, the 24*7 chat support & direct call services are provided to procure the sample report.

1. Introduction
• Market Definition
• Market Segmentation
• Research Methodology

2. Executive Summary
• Key Findings
• Market Overview
•  Market Highlights

3. Market Overview
• Market Size and Growth Potential
• Market Trends
• Market Drivers
• Market Restraints
• Market Opportunities
• Porter’s Five Forces Analysis

4. Morquio Syndrome Treatment Market, By Type of Treatment
• Enzyme Replacement Therapy (ERT)
• Surgical Interventions
• Pain Management
• Physical and Occupational Therapy

5. Morquio Syndrome Treatment Market, By Mode of Administration
• Intravenous (IV) Infusion
• Oral Administration
• Intra-Articular Injection

6. Morquio Syndrome Treatment Market, By End-User
• Hospitals and Clinics
• Specialty Centers
• Home Healthcare
• Rehabilitation Centers

7. Regional Analysis
• North America
• United States
• Canada
• Mexico
• Europe
• United Kingdom
• Germany
• France
• Italy
• Asia-Pacific
• China
• Japan
• India
• Australia
• Latin America
• Brazil
• Argentina
• Chile
• Middle East and Africa
• South Africa
• Saudi Arabia
• UAE

8. Market Dynamics
• Market Drivers
• Market Restraints
• Market Opportunities
• Impact of COVID-19 on the Market

9. Competitive Landscape
• Key Players
• Market Share Analysis

10. Company Profiles

• BioMarin Pharmaceuticals Inc. (USA)
• Ultragenyx Pharmaceutical Inc. (USA)
• Shire plc (Ireland)
• ArmaGen, Inc. (USA)
• Enzyvant Therapeutics Inc. (USA)

• Amicus Therapeutics (US)
• Orchard Therapeutics (US)
• Spark Therapeutics (US)
• MyoGene Inc. (US)
• Abeona Therapeutics Inc. (US)
• Dimension Therapeutics Inc. (US)

11. Market Outlook and Opportunities
• Emerging Technologies
• Future Market Trends
• Investment Opportunities

12. Appendix
• List of Abbreviations
• Sources and References

Report Research Methodology

Research methodology

Verified Market Research uses the latest researching tools to offer accurate data insights. Our experts deliver the best research reports that have revenue generating recommendations. Analysts carry out extensive research using both top-down and bottom up methods. This helps in exploring the market from different dimensions.

This additionally supports the market researchers in segmenting different segments of the market for analysing them individually.

We appoint data triangulation strategies to explore different areas of the market. This way, we ensure that all our clients get reliable insights associated with the market. Different elements of research methodology appointed by our experts include:

Exploratory data mining

Market is filled with data. All the data is collected in raw format that undergoes a strict filtering system to ensure that only the required data is left behind. The leftover data is properly validated and its authenticity (of source) is checked before using it further. We also collect and mix the data from our previous market research reports.

All the previous reports are stored in our large in-house data repository. Also, the experts gather reliable information from the paid databases.

expert data mining

For understanding the entire market landscape, we need to get details about the past and ongoing trends also. To achieve this, we collect data from different members of the market (distributors and suppliers) along with government websites.

Last piece of the ‘market research’ puzzle is done by going through the data collected from questionnaires, journals and surveys. VMR analysts also give emphasis to different industry dynamics such as market drivers, restraints and monetary trends. As a result, the final set of collected data is a combination of different forms of raw statistics. All of this data is carved into usable information by putting it through authentication procedures and by using best in-class cross-validation techniques.

Data Collection Matrix

PerspectivePrimary ResearchSecondary Research
Supplier side
  • Fabricators
  • Technology purveyors and wholesalers
  • Competitor company’s business reports and newsletters
  • Government publications and websites
  • Independent investigations
  • Economic and demographic specifics
Demand side
  • End-user surveys
  • Consumer surveys
  • Mystery shopping
  • Case studies
  • Reference customer

Econometrics and data visualization model

data visualiztion model

Our analysts offer market evaluations and forecasts using the industry-first simulation models. They utilize the BI-enabled dashboard to deliver real-time market statistics. With the help of embedded analytics, the clients can get details associated with brand analysis. They can also use the online reporting software to understand the different key performance indicators.

All the research models are customized to the prerequisites shared by the global clients.

The collected data includes market dynamics, technology landscape, application development and pricing trends. All of this is fed to the research model which then churns out the relevant data for market study.

Our market research experts offer both short-term (econometric models) and long-term analysis (technology market model) of the market in the same report. This way, the clients can achieve all their goals along with jumping on the emerging opportunities. Technological advancements, new product launches and money flow of the market is compared in different cases to showcase their impacts over the forecasted period.

Analysts use correlation, regression and time series analysis to deliver reliable business insights. Our experienced team of professionals diffuse the technology landscape, regulatory frameworks, economic outlook and business principles to share the details of external factors on the market under investigation.

Different demographics are analyzed individually to give appropriate details about the market. After this, all the region-wise data is joined together to serve the clients with glo-cal perspective. We ensure that all the data is accurate and all the actionable recommendations can be achieved in record time. We work with our clients in every step of the work, from exploring the market to implementing business plans. We largely focus on the following parameters for forecasting about the market under lens:

  • Market drivers and restraints, along with their current and expected impact
  • Raw material scenario and supply v/s price trends
  • Regulatory scenario and expected developments
  • Current capacity and expected capacity additions up to 2027

We assign different weights to the above parameters. This way, we are empowered to quantify their impact on the market’s momentum. Further, it helps us in delivering the evidence related to market growth rates.

Primary validation

The last step of the report making revolves around forecasting of the market. Exhaustive interviews of the industry experts and decision makers of the esteemed organizations are taken to validate the findings of our experts.

The assumptions that are made to obtain the statistics and data elements are cross-checked by interviewing managers over F2F discussions as well as over phone calls.

primary validation

Different members of the market’s value chain such as suppliers, distributors, vendors and end consumers are also approached to deliver an unbiased market picture. All the interviews are conducted across the globe. There is no language barrier due to our experienced and multi-lingual team of professionals. Interviews have the capability to offer critical insights about the market. Current business scenarios and future market expectations escalate the quality of our five-star rated market research reports. Our highly trained team use the primary research with Key Industry Participants (KIPs) for validating the market forecasts:

  • Established market players
  • Raw data suppliers
  • Network participants such as distributors
  • End consumers

The aims of doing primary research are:

  • Verifying the collected data in terms of accuracy and reliability.
  • To understand the ongoing market trends and to foresee the future market growth patterns.

Industry Analysis Matrix

Qualitative analysisQuantitative analysis
  • Global industry landscape and trends
  • Market momentum and key issues
  • Technology landscape
  • Market’s emerging opportunities
  • Porter’s analysis and PESTEL analysis
  • Competitive landscape and component benchmarking
  • Policy and regulatory scenario
  • Market revenue estimates and forecast up to 2027
  • Market revenue estimates and forecasts up to 2027, by technology
  • Market revenue estimates and forecasts up to 2027, by application
  • Market revenue estimates and forecasts up to 2027, by type
  • Market revenue estimates and forecasts up to 2027, by component
  • Regional market revenue forecasts, by technology
  • Regional market revenue forecasts, by application
  • Regional market revenue forecasts, by type
  • Regional market revenue forecasts, by component

Morquio Syndrome Treatment Market

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