关键词：法布里病；遗传溶酶体；贮积病；过度；GL-3
摘 要：Fabry disease is an inheritable lysosomal storage disease characterized by excessive accumulation of GL-3 in lysosomes. It results from the deficiency of α-Gal and can result in symptoms such as fatigue, angiokeratoma, tinnitus, kidney failure, heart disorders and anervous system problems. Fabry disease results from mutations in the GLA gene resulting in decreased activity or complete absence of α-Gal.
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. Disease Overview

05.1 Lysosomal Storage Diseases

05.2 Fabry Disease

Etiology and Pathophysiology

Signs and Symptoms

Epidemiology

Inheritance Pattern

06. Introduction

07. Market Landscape

07.1 Market Overview

07.2 Market Size and Forecast

07.3 Historical Analysis of Global Fabry Disease

Market

07.4 Five Forces Analysis

08. Market Segmentation by Cell Line Used

for Manufacturing Drug

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

Key News

Mergers and Acquisitions

17.2 Market Share Analysis 2014

17.3 Other Prominent Vendors and Future Vendors

18. Pipeline Analysis

18.1 Clinical Trial Analysis of Pipeline Candidates

AT1001

GZ402671

PRX-102

Genz-682452

19. Fabry Registry

20. Key Vendor Analysis

20.1 Genzyme

Key Facts

Business Overview

Product Segmentation by Revenue 2013

Product Segmentation by Revenue 2012 and 2013

Geographical Segmentation

Business Strategy

Recent Developments

SWOT Analysis

20.2 Shire

Key Facts

Business Overview

Geographical Segmentation by Revenue 2013

Business Strategy

Recent Developments

SWOT Analysis

21. Other Reports in this Series