Product Code: IRTNTR3860
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?
Table of Contents
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
- 08.1.1. ERTs
- 08.1.2. SRT
- 08.1.3. Cystine-Depletors
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
- 17.2.1. Genzyme
- 17.2.2. Shire
- 17.2.3. BioMarin
- 17.2.4. Actelion
- 18.1. Other Prominent Vendors
19. Key Vendor Analysis
- 19.1. Actelion
- 19.1.1. Business Overview
- 19.1.2. Key Information
- 19.1.3. SWOT Analysis
- 19.2. BioMarin
- 19.2.1. Business Overview
- 19.2.2. Key Information
- 19.2.3. SWOT Analysis
- 19.3. Genzyme
- 19.3.1. Business Overview
- 19.3.2. Key Information
- 19.3.3. SWOT Analysis
- 19.5. Shire
- 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
- Exhibit 5: Global Lysosomal Storage Diseases Market Segmentation by Drug
- Exhibit 6: Global Lysosomal Storage Diseases Market by Geographical
- 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
- Exhibit 10: Geography-wise Revenue of Genzyme Corp's LSD Products in 2013
- Exhibit 11: Geography-wise Revenue of Naglazyme in 2013 (US$ million)