UK Pompe Disease 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. The market for Pompe disease Drugs is growing due to increase in awareness about the disease , better diagnosis, advancements in treatment options and increase in research and development activities by different pharmaceutical companies and research organizations. Sanofi Genzyme ,BioMarin Pharmaceutical Inc., Avrobio Inc., Amicus Therapeutics Inc. are the key market players operating in global pompe disease market.
UK Pompe Disease Drugs Market is valued at around $38.2 Mn in 2022 and is projected to reach $48.4 Mn by 2030, exhibiting a CAGR of 3% during the forecast period 2023-2030.
A genetic mutation that alters the function of the enzyme known as acid alpha-glucosidase (GAA) is what causes Pompe's illness. This enzyme converts glycogen, a kind of sugar that is stored in the body, into glucose, which the body can utilise as fuel. GAA enzyme activity is low or nonexistent in Pompe illness, which causes the buildup of glycogen in many organs, especially muscles. The symptoms of this disease are Muscular weakness, hypotonia, respiratory problems, fatigue and an enlarged heart. Enzyme replacement therapy, which entails giving patients recombinant acid alpha-glucosidase (GAA), the missing enzyme, is the main treatment for Pompe disease. ERT tries to control the disease's symptoms and lessen glycogen buildup. Companies that have created and marketed enzyme replacement therapy for Pompe disease are the major players in the market for medications for the condition and are also projected to grow in future.
Market Drivers
Improve in diagnosis, advancements in treatment options, development of Enzyme replacement therapy drugs and increase in research and development activities by different pharmaceutical companies and research organizations are all market growth drivers.
Market Restraints
limited patient population, high treatment costs, and regulatory complexities associated with the approval of drugs all act as market growth restraints.
Key players
Sanofi Pfizer Shire Alexion Pharmaceuticals CSL Behring Octapharma Grifols Kedrion Biopharma Baxter International GlycoMimetics1. 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
By Treatment type
By Age of Onset
By End-Users
By Distribution Channels:
Methodology for Database Creation
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