LONDON--(BUSINESS WIRE)--Technavio, a market research company, announces their latest report on gene therapy for mucopolysaccharidosis for the period 2018-2022. The market research report provides a detailed study about mucopolysaccharidosis along with the pipeline landscape. It also consists of a comparative analysis and indication analysis, including the major regulatory authorities in different geographies.
Gene therapy for mucopolysaccharidosis - an overview of the market
Mucopolysaccharidosis consists of a group of more than 40 genetic disorders that forms a part of lysosomal storage disease. They are caused by an inherited deficiency of an enzyme involved in the degradation of acid glycosaminoglycans (GAGs), which was previously known as mucopolysaccharidosis. All of the mucopolysaccharidoses are rare, autosomal recessive, except for mucopolysaccharidosis III, as it is X-linked.
There are seven distinct clinical types and a number of subtypes of mucopolysaccharidosis that has been identified based on the enzyme deficiency involved. The report consists of mucopolysaccharidosis I, mucopolysaccharidosis II, mucopolysaccharidosis III A, and mucopolysaccharidosis III B. Out of these, only four are considered for gene therapy.
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Mucopolysaccharidosis has no cure, but the symptoms can be treated instead. The current therapies for mucopolysaccharidosis focus on slowing down the progression of the disease rather than developing a cure.
According to a senior analyst at Technavio for infectious and rare diseases, “Over the past few years, gene therapy has emerged as one of the most exciting approaches for the treatment of mucopolysaccharidosis. One of the promising therapy is replacement gene therapy, which replaces faulty genes with normal ones in the body. It is considered to be a potential cure to mucopolysaccharidoses and a promising option in the long run.”
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Gene therapy for mucopolysaccharidosis – segmentation analysis
The gene therapy for mucopolysaccharidosis is segmented on the basis of RoA, which includes intravenous, ICV, intracerebral, intracisternal, and unspecified (parenteral). Intravenous and ICV held equal market shares of approximately 29%.
The report also consists of companies that are actively involved in the development of stem cell therapy for multiple sclerosis patients. Based on their financial status, the companies are segmented into Tier-1, Tier-2, and Tier-3.
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