Lysosomal storage diseases (LSDs) refer a group of genetic disorders that are caused by the deficiency of the lysosomal enzyme and activator proteins. Lysosomal enzyme helps in hydrolysis of protein, polysaccharide, DNA, RNA and lipid. In addition, lysosome also works as a waste disposal system of the cell by digesting unwanted material such as outside particles and unwanted macromolecules. Deficiency of lysosomal enzyme creates a defect in lysosomal functions, which results undigested material accumulation within the lysosome and causes disorders. Symptoms of lysosomal storage diseases are vary from person to person, it is depends on type of disorder, rate of progression, age of onset and others. Types of lysosomal diseases include gaucher disease, mucopolysaccharidosis, cystinosis, pompe disease and NPC. Some of the treatments for lysosomal storage diseases include enzyme replacement therapy, marrow replanting therapy and genetic therapy. On the basis of drug class global lysosomal storage diseases market can be segmented into ERTs, SRT and cystine-depletors.
North America, followed by Europe, has the largest market for lysosomal storage diseases due to increasing awareness and diagnosis of lysosomal storage diseases in this region. Asia is expected to show high growth rate in the lysosomal storage diseases market in next few years due to government initiatives and increasing awareness about rare diseases in the region.
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Special provisions for rare and orphan diseases, technological advancement, increasing orphan disease research, new therapeutic approaches for genetic diseases and rise in government expenditure are expected to drive the market for global lysosomal storage diseases. In addition, rise in awareness about lysosomal storage diseases through government initiatives are expected to drive the market for lysosomal storage diseases. However, small patient population and huge cost involved in R&D of drug development is a restraining for the growth for global lysosomal storage diseases market.
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Growing demographics and economies in the developing countries such as Japan is expected to offer new opportunities to lysosomal storage diseases market. In addition, innovations in rare diseases therapies and rise in awareness about the orphan diseases are expected to offer new opportunity to global lysosomal storage diseases market. However, increase in generic erosion is a challenge for the global lysosomal storage diseases market. Increasing number of partnerships and collaborations and rise in number of mergers and acquisitions in the market are some of the recent trends that have been observed in global lysosomal storage diseases market. Some of the major companies operating in the global lysosomal storage diseases market are Actelion Pharmaceuticals, Shire, Raptor, BioMarin Pharmaceutical, Genzyme Corporation, Recordati, Pfizer Inc., Sigma-Tau Pharmaceuticals, Inc., Mylan Inc. and Protalix Biotherapeutics.
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