UAE Morquio Syndrome (MPS-IV) Drugs Market Report 2023 to 2030

UAE Morquio Syndrome (MPS-IV) Drugs Market Analysis

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UAE Morquio Syndrome (MPS-IV) Drugs Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 ? 2030. As genetic illnesses like Morquio Syndrome (MPS-IV) has become more common, there is an increasing demand for specialised drugs to treat them. Unmet clinical demands and improved treatment outcomes, Orphan Drug Designation and Incentives, Patient Advocacy and Support programmes. Major global players in Morquio Syndrome (MPS-IV) Drugs Market are BioMarin, Genzyme Corporation, Shire, JCR Pharmaceuticals Co., Ltd., Sanofi, Sangamo Drugs, ArmaGen and GREEN CROSS CORP, Concert Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., and other players.

ID: IN10AERD098 CATEGORY: Rare Diseases GEOGRAPHY: UAE AUTHOR: Nipun Arora

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UAE Morquio Syndrome (MPS-IV) Drugs Market Analysis Summary

UAE Morquio Syndrome (MPS-IV) Drugs Market is valued at around $0.48 Mn in 2022 and is projected to reach $1.08 Mn by 2030, exhibiting a CAGR of 10.54% during the forecast period 2023-2030.

Morquio Syndrome is a rare genetic metabolic disease or birth abnormality that mostly affects the skeleton. It is also known as MPS IV and mucopolysaccharidosis IV. It's important to note that while ERTs can help manage the symptoms of Morquio Syndrome, they do not provide a cure and may not reverse the existing damage caused by the disease. ERTs are typically administered through intravenous infusions and require regular lifelong treatment like Elaprase (idursulfase) is an ERT approved for treating MPS-IV type A (Morquio A syndrome). It provides a synthetic version of the enzyme iduronate-2-sulfatase, which is deficient in individuals with Morquio A syndrome. Vimizim (Elosulfase alfa): Vimizim is an ERT approved for treating MPS-IV type IVA (Morquio A syndrome). It provides a synthetic form of the enzyme N-acetylgalactosamine-6-sulfatase (GALNS), deficient in individuals with Morquio A syndrome. By giving deficient enzyme accumulation of glycosaminoglycans is decreased.

Orthopaedic interventions, Physical therapy and rehabilitation, Respiratory support and pain management are also done for this disease. Drugs like Elaprase and ALDURAZYME must cross the blood-brain barrier to be successful in treating the condition. The most severely affected patients with mucopolysaccharidosis (MPS) are unable to benefit from conventional therapy for their neurological symptoms and problems. For instance, the neurological symptoms of Hunter syndrome, which affect around two-thirds of all patients, are among the most incapacitating in those with the most severe symptoms. A sizeable fraction of the population still requires effective therapy because the neurological symptoms cannot be addressed by the available medications.

Major global players in Morquio Syndrome (MPS-IV) Drugs Market are BioMarin, Genzyme Corporation, Shire, JCR Pharmaceuticals Co., Ltd., Sanofi, Sangamo Drugs, ArmaGen and GREEN CROSS CORP, Concert Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., and other players.

Market Dynamics

Market Drivers

Unmet clinical demands and improved treatment outcomes are major drivers of the increased demand for cutting-edge medications. Orphan Drug Designation and Incentives like exclusivity, tax credits, and research grants. Increase in Research and Development effects and funding, Patient Advocacy and Support programmes. All these are market growth drivers.

Market Restraints

High treatment costs are a defining feature of the Morquio syndrome treatment market. Enzyme replacement therapy is one of the pricey therapies for Morquio syndrome that not all patients may be able to afford.

Limited Treatment Options, Challenges in Diagnosis, Disease Complexity and Heterogeneity, Limited Awareness and Education. All these factors act as market growth restraints.

Key players

BioMarin Pharmaceutical Orchard Therapeutics uniQure Astellas Pharma PTC Therapeutics Pfizer Bluebird Bio Stealth Biotherapeutics Sarepta Therapeutics Amicus Therapeutics

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For UAE Morquio Syndrome (MPS-IV) Drugs Market

By Treatment

Enzyme Replacement Therapy
Others

By Disease Type :

Morquio A
Morquio B

By Distribution Channels :

Hospital Pharmacies
Retail Pharmacies
Online Pharmacies

Methodology for Database Creation

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How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

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Analyzing historical proprietary data collected from multiple projects.
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Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

Searching through academic conferences, published research, citations, and social media platforms
Collecting and compiling diverse data to build a comprehensive and detailed database
Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
Continuously refreshing our database to ensure that the information remains current and reliable.
Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

Merging historical data from secondary research with real-time data from primary research.
Conducting thorough data validation and cleansing to remove inconsistencies and errors.
Organizing data into a structured format that is easily accessible and usable for various applications.
Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.