Global Pompe Disease Market 2015-2019

Category : Pharmaceuticals  | Published Date : Apr-2015 | Pages : 71

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 
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?

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

Publisher Name : Technavio

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