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Global Pompe Disease Market 2015-2019

  • April 2015
  • -
  • Infiniti Research Limited
  • -
  • 71 pages

About Pompe Disease
Pompe disease is a type of LSD. LSD refers to a group of diseases arising from the accumulation of specific substrates. LSDs are progressive and have variable age of onset and clinical symptoms. LSDs are inherited autosomal recessive disorders. Pompe disease has two broad sub-types: infantile-onset Pompe disease and adult-onset Pompe disease. Pompe disease occurs as a result of mutations in the GAA gene. These mutations are responsible for the preventing the action of the enzyme acid alpha-glucosidase, which is responsible for catabolizing glycogen, thereby allowing sugar to accumulate and reach toxic levels in the lysosomes of cells. The buildup of glycogen in certain organs and tissues, mostly in the muscles, hinders the normal functions of organs. Pompe disease is an autosomal recessive disease. This implies that both the parents pass on one abnormally mutated copy of the gene to their child. A parent carrying an abnormal copy of the gene is known as a carrier. Pompe disease affects both men and women, irrespective of the gender. Pompe disease affects people across all ethnicities and races. Individuals affected with Pompe disease acquire the disease at birth, however, the onset of symptoms varies extensively. More than 300 genetic mutations that can cause Pompe disease have been identified by the researchers till date. Though there is no cure for Pompe disease, diet therapy and enzyme replacement therapy are effective in reducing the clinical manifestations of the disease.

TechNavio's analysts forecast the Global Pompe Disease market to grow at a CAGR of 11.79 percent over the period 2014-2019.


Covered in this Report
This report covers the present scenario and the growth prospects of the Global Pompe Disease market for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of drugs available in the market for the management of Pompe disease.

The report also presents the vendor landscape and a corresponding detailed analysis of the major vendors in the Global Pompe Disease market. The report also considers the revenue that is likely to be generated from the drug candidates that are under various phases of development. In addition, it discusses the major drivers that influence the growth of the market, the challenges faced by the vendors and the market at large, as well as the key trends that contribute to the growth the market.

TechNavio's report, Global Pompe Disease Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, and the EMEA and APAC regions; it also covers the Global Pompe Disease market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.


Key Regions
• Americas
• APAC
• EMEA


Key Vendors
• Amicus Therapeutics
• BioMarin Pharmaceutical
• Genzyme

Other Prominent Vendors
• Audentes Therapeutics
• EpiVax
• Oxyrane
• Sangamo BioSciences
• Valerion Therapeutics


Market Driver
• Special Provisions for Orphan Drugs
• For a full, detailed list, view our report


Market Challenge
• Limited Patient Population
• For a full, detailed list, view our report


Market Trend
• Regulatory Assistance in Emerging Nations
• For a full, detailed list, view our report


Key Questions Answered in this Report
• What will the market size be in 2019 and what will the growth rate be?
• What are the key market trends?
• What is driving this market?
• What are the challenges to market growth?
• Who are the key vendors in this market space?
• What are the market opportunities and threats faced by the key vendors?
• What are the strengths and weaknesses of the key vendors?

Table Of Contents

Global Pompe Disease Market 2015-2019
01. Executive Summary
02. List of Abbreviations
03. Scope of the Report
03.1 Market Overview
03.2 Product Offerings
04. Market Research Methodology
04.1 Market Research Process
04.2 Research Methodology
05. Introduction
06. Disease Overview
06.1 Understanding the Disease
06.1.1 Types of Pompe Disease
06.1.2 Symptoms
06.1.3 Diagnosis
06.2 Etiology and Pathogenesis
06.2.1 Biochemical Pathway
06.3 Epidemiology
06.4 Management
06.5 Economic Burden
07. Market Landscape
07.1 Market Overview
07.2 Market Size and Forecast
07.3 Five Forces Analysis
08. Pipeline Analysis
08.1 Key Information on Pipeline Candidates
08.1.1 rAAV1-CMV-GAA
08.1.2 OXY-2810
08.1.3 Tregitopes for Pompe Disease
08.1.4 AT-002
08.1.5 ZFP Transcription Factors for LSDs
08.1.6 VAL-1221
09. Geographical Segmentation
10. Buying Criteria
11. Market Growth Drivers
12. Drivers and their Impact
13. Market Challenges
14. Impact of Drivers and Challenges
15. Market Trends
16. Trends and their Impact
17. Vendor Landscape
17.1 Competitive Scenario
17.1.1 Key News
17.1.2 Mergers and Acquisitions
17.2 Market Share Analysis 2014
17.2.1 Genzyme
17.2.2 BioMarin Pharmaceutical
17.2.3 Amicus Therapeutics
17.3 Other and Future Prominent Vendors
18. Key Vendor Analysis
18.1 Amicus Therapeutics
18.1.1 Key Facts
18.1.2 Business Overview
18.1.3 Business Strategy
18.1.4 Key Developments
18.1.5 SWOT Analysis
18.2 BioMarin Pharmaceutical
18.2.1 Key Facts
18.2.2 Business Overview
18.2.3 Segmentation by Revenue 2014
18.2.4 Segmentation by Revenue 2013 and 2014
18.2.5 Geographical Segmentation by Revenue 2014
18.2.6 Business Strategy
18.2.7 Recent Developments
18.2.8 SWOT Analysis
18.3 Genzyme
18.3.1 Key Facts
18.3.2 Business Overview
18.3.3 Product Segmentation by Revenue 2013
18.3.4 Product Segmentation by Revenue 2012 and 2013
18.3.5 Geographical Segmentation
18.3.6 Business Strategy
18.3.7 Recent Developments
18.3.8 SWOT Analysis
19. Other Reports in this Series


List of Exhibits:
Exhibit 1: Market Research Methodology
Exhibit 2: Snapshot of Pompe Disease
Exhibit 3: Heredogram of Pompe Disease
Exhibit 4: Types of Pompe Disease
Exhibit 5: Signs and Symptoms of Pompe Disease
Exhibit 6: Differential Diagnosis of Pompe Disease in Infants
Exhibit 7: Differential Diagnosis of Pompe Disease in Children and Adults
Exhibit 8: Biochemical Pathway of Pompe Disease
Exhibit 9: Approaches to Treat Pompe Disease
Exhibit 10: Economic Burden of Pompe Disease
Exhibit 11: Snapshot of Global Pompe Disease Market 2014
Exhibit 12: Global Pompe Disease Market 2014-2019 (US$ million)
Exhibit 13: Drivers and Challenges of Global Pompe Disease Market
Exhibit 14: Pipeline Portfolio of Global Pompe Disease Market
Exhibit 15: Segmentation of Global Pompe Disease Market by Geography 2014
Exhibit 16: Genzyme: Product Portfolio
Exhibit 17: BioMarin Pharmaceutical: Segmentation by Revenue 2014
Exhibit 18: BioMarin Pharmaceutical: Segmentation by Revenue 2013 and 2014 (US$ million)
Exhibit 19: BioMarin Pharmaceutical: Geographical Segmentation by Revenue 2014
Exhibit 20: Genzyme: Product Segmentation by Revenue 2013
Exhibit 21: Genzyme: Product Segmentation by Revenue 2012 and 2013 (US$ million)
Exhibit 22: Genzyme: Geographical Segmentation

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