Australia 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.
Australia Morquio Syndrome (MPS-IV) Drugs Market is valued at around $1.14 Mn in 2022 and is projected to reach $2.64 Mn by 2030, exhibiting a CAGR of 11.04% 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.
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.
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 playersBioMarin Pharmaceutical Inc. Pfizer Sanofi Shire plc Catalyst Biosciences uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Intercept Pharmaceuticals Novartis
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)
220.127.116.11 Product Applications and Services
18.104.22.168 Recent Developments
22.214.171.124 Partnerships Ecosystem
126.96.36.199 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
By Disease Type :
By Distribution Channels :
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