Lysosomal storage disease (LTD) is caused by defect in lysosomal functions and degradation pathway. It is caused by progressive accumulation of metabolites. LTD is a rare inherited metabolic disorder. There are 50 types of lysosomal storage diseases. Lysosomes are organelle present in eukaryotic cell. These consist of degradative enzymes enclosed in a membrane. A study conducted by Mayo Clinic in the U.S. indicated that the incidence of lysosomal disorder ranges between 1 in 4000 and 1 in 13,000 live births. Tay-Sachs disease was the first lysosomal disorder to be diagnosed in 1881.
Increase in incidence of lysosomal diseases, rise in diagnostic rate, surge in awareness about this rare disorder, increase in focus on research and development for diagnosis & drug development for treatment of lysosomal diseases, and implementation of orphan drug regulation that provides marketing and commercial benefits to pharmaceutical companies fuel the growth of the global lysosomal storage disease treatment market. However, high cost of available treatment, underdiagnosed lysosomal disorders because of heterogeneity of the rare lysosomal disorder, and decrease in efficiency of treatment option are likely to hamper the growth of the global lysosomal storage disease treatment market. Availability and advancement of chaperone and gene therapy is expected to propel the market during the forecast period.
The global lysosomal storage disease treatment market can be segmented based on disease type, therapy, end-user, and region. In terms of disease type, the global market can be classified into aspartylglucosaminuria, Batten disease, cystinosis, Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, and others. Gaucher disease is the most common type of lysosomal disorder which is characterized by neurological complication. Tay-Sachs disease is another common lysosomal disorder propelling the global lysosomal storage disease treatment market.
Based on therapy, the global lysosomal storage disease treatment market can be categorized into enzyme replacement therapy, stem cell transplantation, substrate reduction therapy, and others. Enzyme replacement is the most preferred therapy for the treatment of lysosomal disorder. It is effective in reducing toxic storage material and organomegaly. Enzyme replacement therapy also helps in improving organ function in patients suffering from lysosomal disorder. Advanced research in stem cell transplantation has increased the success rate of treatment of lysosomal disease. Rise in the number of stem cell banks also fuels the growth of the global lysosomal storage disease treatment market.
In terms of end-user, the global lysosomal storage disease treatment market can be divided into hospitals, clinics, stem transplant centers, research organizations, and others. Hospitals was the leading segment of the global lysosomal storage disease treatment market in 2017. Research organizations are being funded by governments as well as pharmaceutical companies for the development of new treatment methods and drugs. Hence, the research organizations segment is expected to witness significant growth during the forecast period.
In terms of region, the global lysosomal storage disease treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is a leading market for lysosomal disease treatment because of strong health care, economic, and financial infrastructure. Europe is a major hub for health care research and development. Moreover, high per capita income of the people is attributed to the growth of the global lysosomal storage disease disorder treatment market in the region. Asia Pacific consists of developing countries with high prevalence of inherited abnormalities and disorders. Hence, the market in the region is expected to grow at a rapid pace during the forecast period.
Key players operating in the global lysosomal storage disease treatment market include Shire, Pfizer, Inc., Sanofi, BioMarin, Merck & Co., Inc., Actelion Pharmaceuticals Ltd., and Eli Lilly and Company.
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