The Pompe Disease market report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Pompe Disease (PD) from 2018 to 2030 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.
Pompe Disease Overview
Pompe disease (PD), also known as glycogen storage disease type II (GSDII) or “acid maltase deficiency”, is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the α-1, 4- and α-1, 6-glycosidic bonds of glycogen to glucose. The disease is caused pathogenic variations in the acid alpha-glucosidase (GAA) gene. Close to 500 different GAA gene variations have been identified in families with this disorder.
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List of regions covered in the report
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Pompe Disease Diagnosis
The diagnosis of Pompe disease often poses a diagnostic dilemma due to the rarity of the condition and the relatively nonspecific nature of the phenotypic features that may only lead to suspicion of Pompe disease. A chest x-ray and electrocardiogram (ECG) are valuable screening tests in the diagnostic algorithm for infantile Pompe disease and an echocardiogram is a valuable next step. Chest x-ray shows massive cardiomegaly. ECG shows a short PR interval as well as very tall QRS complexes. Among patients presenting with cardiomyopathy, an electromyogram (EMG) can be useful to document presymptomatic myopathy. Blood tests should include a serum creatine kinase (CK) examination. CK elevation is a sensitive although very nonspecific marker for Pompe disease.
Pompe Disease Treatment Market
The treatment of Pompe disease is disease-specific, symptomatic, and supportive. Treatment requires the coordinated efforts of a team of specialists with expertise in treating neuromuscular disorders. Pediatricians or internists, neurologists, orthopedists, cardiologists, dieticians, and other healthcare professionals may need to systematically and comprehensively plan an affect child’s treatment. Enzyme replacement therapy is an approved treatment for all patients with Pompe disease. It involves the intravenous administration of recombinant human acid α-glucosidase. This treatment, manufactured by Genzyme, a Sanofi Corporation.
Pompe Disease Market Insights
Although historically, Supportive Care had been the mainstay of treatment (thereby generating a massive amount of revenue), but the approval and subsequent launch of ERT in 2006 after decades of research, helped turn the treatment landscape upside down, prompting a new era in the treatment of this disease. This was the first disease-specific treatment for an inherited muscular disorder consisting of the intravenous administration of recombinant human GAA (Myozyme/Lumizyme).
Pompe Disease Emerging Drugs
Pompe Disease Marketed Drugs
Pompe Disease Market Outlook
The Pompe Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
Pompe Disease Market Report Scope
Table of Content
1. Key Insights
2. Executive Summary of Pompe Disease (PD)
3. SWOT Analysis for Pompe Disease
4. Pompe Disease Market Overview at a Glance
5. Disease Background and Overview
6. Epidemiology and Patient Population
7. Country Wise-Epidemiology of Pompe Disease
8. Treatment and Management of Pompe Disease
9. Gene Therapy: What Future Holds
10. Case Study
11. Patient Journey
12. Unmet Needs
13. Marketed Products
14. Emerging Therapies
15. Other Promising Candidates
16. Pompe Disease: 7 Major Market Analysis
17. Market Outlook by Country
18. Market Drivers
19. Market Barriers
20. Health Economics of Pompe Disease
21. Market access and Reimbursement
22. Appendix
23. DelveInsight Capabilities
24. Disclaimer
25. About DelveInsight
Pompe Disease Market Report Highlights
About Us
DelveInsight is a Business Consulting and Market research company, providing expert business solutions for life science vertical and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.
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