About Lysosomal Storage Diseases
The term lysosomal storage diseases refers to a group of rare inherited disorders caused by the deficiency of lysosomal enzymes, activator proteins, proteins required for normal post-translational modification of lysosomal enzymes or proteins required for intracellular transport between lysosome and other cellular compartments. These deficiencies result in deficient enzymatic activity which, in turn, leads to accumulation of partially digested or undigested macromolecules inside the cell. The clinical manifestations of lysosomal storage diseases depend on the type of substrate stored, cell types affected by this storage, and resulting organ involvement. Due to the high variability of these components, the clinical manifestations of even a single disease vary from person to person. These symptoms also vary between members of the same family with identical mutations. The age of onset also varies for lysosomal storage diseases depending on the rate of substrate accumulation. Lysosomal storage diseases are classified on the basis of the substrate being accumulated or on the type of molecular defect associated with the disease. Neuronal ceroid-lipofuscinoses vary from classical lysosomal storage diseases where the component that gets accumulated is mitochondrial ATP synthase subunit C or sphingolipid activator proteins A and D.
TechNavio's analysts forecast the Global Lysosomal Storage Diseases market will grow at a CAGR of 10.01 percent over the period 2013-2018.
Covered in this Report
This report covers the present scenario and the growth prospects of the Global Lysosomal Storage Diseases market for the period 2014-2018. To calculate the market size, the report considers revenue from the sales of various drugs available in the market for the management of different types of lysosomal storage diseases, which include:
• Gaucher disease
• Fabry disease
• Pompe disease
TechNavio's report, the Global Lysosomal Storage Diseases Market 2014-2018, 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 Lysosomal Storage Diseases market landscape and its growth prospects in the coming years. The report also includes a discussion of the key vendors operating in this market.
• Actelion Pharmaceuticals Ltd.
• BioMarin Pharmaceutical Inc.
• Genzyme Corp.
• Shire plc
Other Prominent Vendors
Key Market Driver
• Special Provisions for Orphan Drugs
• For a full, detailed list, view our report.
Key Market Challenge
• Increase in Generic Erosion
• For a full, detailed list, view our report.
Key Market Trend
• Fierce Market Competition
• Increasing Use of Statins.
Key Questions Answered in this Report
• What will the market size be in 2018 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
06. Disease Overview
07. Market Landscape
07.1 Market Overview
07.2 Market Size and Forecast
07.3 First Approved Drugs for Treatment of Lysosomal Storage Diseases
07.4 Five Forces Analysis
08. Market Segmentation by Drug Class
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 2013
18.1 Other Prominent Vendors
19. Key Vendor Analysis
19.1.1 Business Overview
19.1.2 Key Information
19.1.3 SWOT Analysis
19.2.1 Business Overview
19.2.2 Key Information
19.2.3 SWOT Analysis
19.3.1 Business Overview
19.3.2 Key Information
19.3.3 SWOT Analysis
19.5.1 Business Overview
19.5.2 Key Information
19.5.3 SWOT Analysis
20. Other Reports in this Series
List of Exhibits
Exhibit 1: Market Research Methodology
Exhibit 2: Classification of Lysosomal Storage Diseases
Exhibit 3: Global Lysosomal Storage Diseases Market 2013-2018 (US$ billion)
Exhibit 4: Global Lysosomal Storage Diseases Market Segmentation by Drug Class
Exhibit 5: Global Lysosomal Storage Diseases Market Segmentation by Drug Class 2013
Exhibit 6: Global Lysosomal Storage Diseases Market by Geographical Segmentation 2013
Exhibit 7: Market Revenue for Major Drugs 2013
Exhibit 8: Percentage Revenue Share of LSD Drugs of Genzyme 2013
Exhibit 9: Geography-wise Revenue of Genzyme Corp.’s LSD Products in 2013 (US$ million)
Exhibit 10: Geography-wise Revenue of Genzyme Corp’s LSD Products in 2013
Exhibit 11: Geography-wise Revenue of Naglazyme in 2013 (US$ million)