Boston, MA -- (SBWIRE) -- 08/19/2014 -- 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.

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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
- Mucopolysaccharidosis
- Pompe disease
- Cystinosis
- NPC

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.

Key Regions:

- Americas
- EMEA
- APAC

Key Vendors:

- Actelion Pharmaceuticals Ltd.
- BioMarin Pharmaceutical Inc.
- Genzyme Corp.
- Shire plc

Other Prominent Vendors:

- Mylan
- Pfizer
- Protalix
- Raptor
- Recordati
- Sigma-Tau

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?

Companies Mentioned in this Report: Actelion Pharmaceuticals Ltd., BioMarin Pharmaceutical Inc., Genzyme Corp., Shire plc, Mylan, Pfizer, Protalix, Raptor, Recordati, Sigma-Ta

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