关键词：高雪氏症病;脂肪细胞;脂醣脂类物质;巨噬细胞;骨髓;血小板;骨骼异常
摘 要：Gaucher disease is a genetic disorder characterized by the accumulation of fatty substances in cells and certain organs of the body. It is caused by the deficiency of the enzyme glucocerebrosidase. This results in the accumulation of certain detrimental lipids in the body,especially the glycolipid glucocerebroside. The lipids get accumulated within the macrophages,blood cells, spleen, liver, and bone marrow. Symptoms varies among individuals, with some experiencing no symptoms while others experience severe complications. Hepatosplenomegaly,anemia, thrombocytopenia, and skeletal abnormalities are also reported.
目 录：

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 Gaucher Disease

06.1.2 Symptoms

06.1.3 Diagnosis

06.2 Etiology

06.3 Epidemiology

06.4 Clinical Manifestations

06.5 Management

06.5.1 Enzyme Replacement Therapy

06.5.2 Substrate Reduction Therapy

06.6 Economic Burden

07. Pipeline Analysis

08. Market Landscape

08.1 Market Overview

08.2 Market Size and Forecast

08.3 Five Forces Analysis

09. Market Segmentation by Type of Therapy

09.1 Enzyme Replacement Therapy

09.2 Substrate Reduction Therapy

10. Market Segmentation by Type of Molecule

10.1 Biologics

10.2 Small Molecules

11. Market Segmentation by Route of

Administration

11.1 Oral

11.2 Parenteral

12. Market Segmentation by Dosage Form

12.1 Solid

12.2 Liquid

13. Geographical Segmentation

14. Buying Criteria

15. Market Growth Drivers

16. Drivers and their Impact

17. Market Challenges

18. Impact of Drivers and Challenges

19. Market Trends

20. Trends and their Impact

21. Vendor Landscape

21.1 Competitive Scenario

21.1.1 Key News

21.1.2 Mergers and Acquisitions

21.2 Market Share Analysis 2014

21.2.1 Genzyme

21.2.2 Shire

21.2.3 Actelion Pharmaceuticals

21.2.4 Pfizer

21.3 Other and Future Prominent Vendors

22. Key Vendor Analysis

22.1 Actelion Pharmaceuticals

22.1.1 Key Facts

22.1.2 Business Overview

22.1.3 Geographical Segmentation 2013

22.1.4 Business Strategy

22.1.5 Recent Developments

22.1.6 SWOT Analysis

22.2 Genzyme

22.2.1 Key Facts

22.2.2 Business Overview

22.2.3 Product Segmentation by Revenue 2013

22.2.4 Product Segmentation by Revenue 2012 and 2013

22.2.5 Geographical Segmentation

22.2.6 Business Strategy

22.2.7 Recent Developments

22.2.8 SWOT Analysis

22.3 Pfizer

22.3.1 Key Facts

22.3.2 Business Overview

22.3.3 Business Segmentation by Revenue 2013

22.3.4 Business Segmentation by Revenue 2012 and 2013

22.3.5 Geographical Segmentation by Revenue

22.3.6 Business Strategy

22.3.7 Key Developments

22.3.8 SWOT Analysis

22.4 Shire

22.4.1 Key Facts

22.4.2 Business Overview

22.4.3 Geographical Segmentation by Revenue 2013

22.4.4 Business Strategy

22.4.5 Recent Developments

22.4.6 SWOT Analysis

23. Other Reports in this Series